Category Archives: Genetic Therapy

Global Gaucher Disease Treatment Marketis estimated to surpass 956.2 USD Million mark in 2018 and reach XX USD Million by 2026, at a Compound Annual Growth Rate (CAGR) of XX % during the forecast period 2018-2026 globally.Global Gaucher Disease Treatment Market, By Therapy TypeGaucher disease is a disorder of gene or a hereditary condition in which body fat (lipid) a glucosylceramide gets accumulated into tissue and certain organs, an accumulation is mainly caused by a deficiency of enzyme B-glucocerebrosidase, which then cause complications and is characterized by low blood platelet count, fatigue, bruising, etc. The Gaucher disease is also characterized by the enlargement of the liver and spleen. Sometimes, people may have no disease symptoms and children are more prone to Gaucher disease than adults.

Global Gaucher Disease Treatment Market is driven by rising demand for adequate and well control treatment for the disease. New drug approvals for disease treatment are expected to positively impact global Gaucher disease treatment market in near future. On the contrary, the availability of low patient pool to carry out enough clinical trials is hampering the Global Gaucher Disease Treatment Market.

Global Gaucher Disease Treatment Market has been segmented into disease type, therapy type, distribution channel, and geography. Based on disease type, type 1 gaucher disease, accounted for largest share of the market in 2017, compared to type 2 gaucher disease, and type 3 gaucher disease due to the slight high prevalence over the population. Among the Therapy types, enzyme replacement therapy coupled with recent advancement leading the market segment.

On the basis of region, Global Gaucher disease treatment market is divided into North America, Europe, Asia Pacific, Latin America, and Middle East & Africa. In the year 2018, North America is expected to hold the largest share of the Global Gaucher Disease Treatment Market due to the increasing research activities for drug development. Also, the presence of key market players in the region and their revenue-generating strategies driving the growth of the regional market. European gaucher disease treatment market is expected to remain the second largest market due to increasing awareness programs about gaucher disease treatment.

Maximize Market Research has comprehensively analysed Global Gaucher Disease Treatment Market emphasizing on each and every segment keeping global and regional dynamics in perspective. The driving forces, as well as considerable restraints, have been explained in depth to attain a balanced scenario. The report classifies Global Gaucher disease treatment market into various segments such as disease type, therapy type, distribution channel and regions providing a thorough understanding of the gaucher disease treatment. Ecosystem. Importantly, the report delivers forecasts of the market, giving an insight into the future opportunities that exist in the Global Gaucher Disease Treatment Market.

The objective of the report is to present comprehensive Global Gaucher Disease Treatment Market including all the stakeholders of the industry. The past and current status of the industry with forecasted market size and trends are presented in the report with analysis of complicated data in simple language. The report covers all the aspects of industry with dedicated study of key players that includes market leaders, followers and new entrants by region. PORTER, SVOR, PESTEL analysis with the potential impact of micro-economic factors by region on the market have been presented in the report. External as well as internal factors that are supposed to affect the business positively or negatively have been analyzed, which will give clear futuristic view of the industry to the decision makers.

The report also helps in understanding Global Gaucher Disease Treatment Market North America for Asia Pacific dynamics, structure by analyzing the market segments, and project the Global Gaucher Disease Treatment Market North America for Asia Pacific size. Clear representation of competitive analysis of key players by type, price, financial position, product portfolio, growth strategies, and regional presence in the Global Gaucher Disease Treatment Market North America for Asia Pacific make the report investors guide.Scope of the Global Gaucher Disease Treatment Market:

Global Gaucher Disease Treatment Market, By Disease Type:

Type 1 Type2 Type 3Global Gaucher Disease Treatment Market, By Therapy Type:

Enzyme Replacement Therapy (ERT) Substrate Reduction Therapy (SRT)Global Gaucher Disease Treatment Market, By Region:

North America Europe Asia Pacific Middle East & Africa South AmericaKey Players Operating In Global Gaucher Disease Treatment Market:

Pfizer, Inc. Abbott GlaxoSmithKline (GSK) Genzyme Corporation Eli Lilly & Company Aptalis Pharma Actelion Pharmaceuticals Ltd. Shire Human Genetic Therapies, Inc. Enobia Pharma Inc. Greenovation Biopharma BioMarin Pharmaceutical Inc. Zymenex A/S MedPro Rx JCR Pharmaceuticals Co.Ltd

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Global Gaucher Disease Treatment Market Analysis and Forecast (2019-2026) by Disease Type, by Therapy Type, by Distribution Channel, and by...

Basel, 7 September 2020 - Roche (SIX: RO, ROG; OTCQX: RHHBY) today announced that the United States (US) Food and Drug Administration (FDA) has approved Gavreto (pralsetinib) for the treatment of adults with metastatic rearranged during transfection (RET) fusion-positive non-small cell lung cancer (NSCLC) as detected by an FDA approved test. This indication was approved under the FDAs Accelerated Approval programme, based on data from the phase I/II ARROW study. Continued approval for this indication may be contingent upon verification and description of clinical benefit in a confirmatory trial. Gavreto is a once-daily, oral precision therapy designed to selectively target RET alterations, including fusions and mutations. It is jointly commercialised by Genentech, a wholly owned member of the Roche Group, and Blueprint Medicines in the US and will be commercialised by Roche outside of the US, excluding Greater China*.

The FDA approval of Gavreto for RET fusion-positive non-small cell lung cancer is an important step towards our goal of providing an effective treatment option for every person diagnosed with lung cancer, no matter how rare or hard-to-treat their type of disease, said Levi Garraway, M.D., Ph.D., Roches Chief Medical Officer and Head of Global Product Development. We remain committed to finding personalised treatment options for people with cancer based on specific genomic or molecular alterations, and we look forward to partnering with Blueprint Medicines to further explore the potential of Gavreto across multiple RET-altered tumour types.

RET-activating fusions and mutations are key disease drivers in many cancer types, including NSCLC and medullary thyroid cancer (MTC), and treatment options that selectively target these genetic alterations are limited. In NSCLC, RET fusions represent approximately 1-2% of patients.1 Biomarker testing for these fusions is the most effective way to identify people who are eligible for treatment with Gavreto.

The approval is based on the results from the phase I/II ARROW study, in which Gavreto produced durable clinical responses in people with RET fusion-positive NSCLC with or without prior therapy, and regardless of RET fusion partner or central nervous system involvement.2 Gavreto demonstrated an overall response rate (ORR) of 57% (95% CI: 46%, 68%) and complete response (CR) rate of 5.7% in the 87 people with NSCLC previously treated with platinum-based chemotherapy, and the median duration of response (DoR) was not reached (95% CI: 15.2 months, not reached).2 In the 27 people with treatment-nave NSCLC, the ORR was 70% (95% CI: 50%, 86%), with an 11% CR rate.2 The most common adverse reactions (25%) were fatigue, constipation, musculoskeletal pain and increased blood pressure (hypertension).2

Gavreto is now the sixth FDA-approved medicine in Roches portfolio of treatments for lung cancer. The FDA granted Breakthrough Therapy Designation to Gavreto for the treatment of RET fusion-positive NSCLC that has progressed following platinum-based chemotherapy and for RET mutation-positive MTC that requires systemic treatment and for which there are no acceptable alternative treatments.

The FDA has also granted Priority Review to Gavreto for the treatment of people with advanced or metastatic RET-mutant MTC and RET fusion-positive thyroid cancer, and is expected to make a decision on approval by 28 February 2021. This New Drug Application (NDA) was accepted for review under the FDA's Real-Time Oncology Review (RTOR) pilot programme, which aims to explore a more efficient review process to ensure safe and effective treatments are available to patients as early as possible.

About the ARROW study3ARROW (NCT03037385) is a phase I/II, open-label, first-in-human study designed to evaluate the safety, tolerability and efficacy of Gavreto, administered orally in people with rearranged during transfection (RET) fusion-positive non-small cell lung cancer (NSCLC), RET-mutant medullary thyroid cancer (MTC), RET fusion-positive thyroid cancer and other RET-altered solid tumours. The trial consists of two parts: a dose escalation portion, which is complete, and an expansion portion in people treated with 400 mg of Gavreto, once-daily. ARROW is being conducted at multiple sites across the United States, European Union and Asia.

About GavretoGavreto is a once-daily, oral precision therapy designed to selectively target RET alterations, including fusions and mutations, regardless of the tissue of origin. Preclinical data have shown that Gavreto inhibits primary RET fusions and mutations that cause cancer in subsets of patients, as well as secondary RET mutations predicted to drive resistance to treatment. Blueprint Medicines and Roche are also co-developing Gavreto for the treatment of patients with various types of RET-altered thyroid cancers and other solid tumours.

About Roche in lung cancerLung cancer is a major area of focus and investment for Roche, and we are committed to developing new approaches, medicines and tests that can help people with this deadly disease. Our goal is to provide an effective treatment option for every person diagnosed with lung cancer. We currently have six approved medicines to treat certain kinds of lung cancer and more than ten medicines being developed to target the most common genetic drivers of lung cancer or to boost the immune system to combat the disease.About RocheRoche is a global pioneer in pharmaceuticals and diagnostics focused on advancing science to improve peoples lives. The combined strengths of pharmaceuticals and diagnostics under one roof have made Roche the leader in personalised healthcare a strategy that aims to fit the right treatment to each patient in the best way possible.

Roche is the worlds largest biotech company, with truly differentiated medicines in oncology, immunology, infectious diseases, ophthalmology and diseases of the central nervous system. Roche is also the world leader in in vitro diagnostics and tissue-based cancer diagnostics, and a frontrunner in diabetes management.

Founded in 1896, Roche continues to search for better ways to prevent, diagnose and treat diseases and make a sustainable contribution to society. The company also aims to improve patient access to medical innovations by working with all relevant stakeholders. More than thirty medicines developed by Roche are included in the World Health Organization Model Lists of Essential Medicines, among them life-saving antibiotics, antimalarials and cancer medicines. Moreover, for the eleventh consecutive year, Roche has been recognised as one of the most sustainable companies in the Pharmaceuticals Industry by the Dow Jones Sustainability Indices (DJSI).

The Roche Group, headquartered in Basel, Switzerland, is active in over 100 countries and in 2019 employed about 98,000 people worldwide. In 2019, Roche invested CHF 11.7 billion in R&D and posted sales of CHF 61.5 billion. Genentech, in the United States, is a wholly owned member of the Roche Group. Roche is the majority shareholder in Chugai Pharmaceutical, Japan. For more information, please visit http://www.roche.com.

All trademarks used or mentioned in this release are protected by law.* Greater China encompasses Mainland China, Hong Kong, Macau and TaiwanReferences[1] Drilon et al. Targeting RET-driven cancers: lessons from evolving preclinical and clinical landscapes. Nat Rev Clin Oncol. 2018;15:15167.[2] Blueprint Medicines. Data on file.[3] ClinicalTrials.gov. Phase 1/2 Study of the Highly-selective RET Inhibitor, Pralsetinib (BLU-667), in Patients With Thyroid Cancer, Non-Small Cell Lung Cancer, and Other Advanced Solid Tumors (ARROW) [Internet; cited August 2020]. Available from: https://clinicaltrials.gov/ct2/show/NCT03037385.

Roche Group Media RelationsPhone: +41 61 688 8888 / e-mail: media.relations@roche.com

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Roche announces FDA approval of Gavreto (pralsetinib) for the treatment of adults with metastatic RET fusion-positive non-small cell lung cancer -...

DUBLIN, Sept. 4, 2020 /PRNewswire/ -- The "Global Gene Therapy Market (by Cell Type, Vector Type, Application, End-User & Region): Insights & Forecast with Potential Impact of COVID-19 (2020-2024)" report has been added to ResearchAndMarkets.com's offering.

The global gene therapy market is expected to reach US$ 6.42 billion in 2024, witnessing growth at a CAGR of 19.29%, over the period 2020-2024. Growth in the gene therapy market has accrued due to the increasing prevalence of chronic diseases, rising healthcare expenditure, expanding urbanization, growth of gene therapy clinical trials and upsurge in economic growth. The market is anticipated to experience certain trends like rapid adoption of personalized medicine, growing occurrence of genetic disorders, advancements in gene therapy and increasing R&D funding. The growth of the market would be challenged by side effects of gene therapy and ethical and safety concerns and high cost of the treatment.

The global gene therapy market has been segmented on the basis of cell type, vector type, application, end-user and region. Depending on the cell type, the market can be bifurcated into somatic cell gene therapy and germ cell gene therapy. According to the vector type, the global gene therapy market can be categorized into retrovirus & gammaretrovirus, adeno-associated viruses (AAV), lentivirus, adenovirus, modified herpes simplex virus and non-viral plasmid vector. Whereas, on the basis of application, the market can be split into oncological disorders, neurological disorders, infectious diseases, cardiovascular diseases, rare diseases and others. Further, in terms of end-user, the global gene therapy market can broadly be segmented into hospitals, specialty treatment centers and other end-users.

The fastest-growing regional market is North America due to the rising incidence of cancer and other target diseases, increasing favorable reimbursement scenario in the region and improvements in healthcare infrastructure. Further, the sudden outbreak of COVID-19 is causing an adverse disruption on the overall economy and society, affecting the rate of gene therapy procedures and clinical trials, which is expected to negatively impact the growth of the global gene therapy market during the forecasted period.

Scope of the report:

Key Target Audience:

Key Topics Covered:

1. Market Overview1.1 Introduction1.2 Diseases Treated by Gene Therapy1.3 Process of Gene Therapy1.4 Types of Gene Therapy1.5 Application Areas for Gene Therapy1.6 Gene Therapy Techniques1.7 Advantages & Disadvantages of Gene Therapy

2. Impact of COVID-192.1 Economic Impact2.2 Decline in Global GDP2.3 Decline in Industrial Production2.4 Impact on Gene Therapy2.5 Impact on Clinical Trials of Gene Therapy

3. Global Market Analysis3.1 Global Gene Therapy Market by Value3.2 Global Gene Therapy Market Forecast by Value3.3 Global Gene Therapy Market by Cell Type3.4 Global Gene Therapy Market by Vector Type3.5 Global Gene Therapy Market by Application3.6 Global Gene Therapy Market by End-User3.7 Global Gene Therapy Market by Region

4. Regional Market Analysis4.1 North America4.2 Europe4.3 Asia Pacific4.4 RoW

5. Market Dynamics5.1 Growth Drivers5.1.1 Increasing Prevalence of Chronic Diseases5.1.2 Rising Healthcare Expenditure5.1.3 Expanding Urbanization5.1.4 Growth of Gene Therapy Clinical Trials5.1.5 Upsurge in Economic Growth5.2 Key Trends and Developments5.2.1 Rapid Adoption of Personalized Medicines5.2.2 Growing Occurrence of Genetic Disorders5.2.3 Advancements in Gene Therapy5.2.4 Increasing R&D Funding5.3 Challenges5.3.1 Side Effects of Gene Therapy5.3.2 Ethical and Safety Concerns5.3.3 High Cost of Treatment

6. Competitive Landscape6.1 Global Market6.1.1 Revenue Comparison of Key Players6.1.2 Market Capitalization Comparison of Key Players6.1.3 R&D Comparison of Key Players

7. Company Profiles

For more information about this report visit https://www.researchandmarkets.com/r/7ewray

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Global Gene Therapy Market is Forecast to Reach US$ 6.42 billion in 2024, Witnessing Growth at a CAGR of 19.29% Over 2020-2024 - WFMZ Allentown

The success of the approved gene therapies has led to an upward surge in the interest of biopharmaceutical developers in this field, resulting in a significant boost in clinical research initiatives and several high value acquisitions

Roots Analysis has announced the addition of Gene Therapy Market (3rd Edition), 2019-2030 report to its list of offerings.

Encouraging clinical results across various metabolic, hematological and ophthalmic disorders have inspired research groups across the world to focus their efforts on the development of novel gene editing therapies. In fact, the gene therapy pipeline has evolved significantly over the past few years, with three products being approved in 2019 alone; namely Beperminogene perplasmid (AnGes), ZOLGENSMA (AveXis) and ZYNTEGLO (bluebird bio). Further, there are multiple pipeline candidates in mid to late-stage (phase II and above) trials that are anticipated to enter the market over the next 5-10 years.

To order this 550+ page report, which features 190+ figures and 355+ tables, please visit this link

Around 470 gene therapies are currently under developmentNearly 45% of pipeline drugs are in the clinical phase, while rest are in the preclinical / discovery stage. Gene augmented therapies presently represent 66% of the total number of such interventions that are in the pipeline. It is worth mentioning that majority of such product candidates are being developed as in vivo gene therapies.

More than 30% of clinical stage pipeline therapies are being designed for treating oncological disordersConsidering the overall pipeline, over 20% of product candidates are being developed to treat various types of cancers, followed by those intended for the treatment of metabolic (15%) and ophthalmic disorders (12%). It is also worth highlighting that adenovirus vectors are presently the preferred vehicles used for the delivery of anticancer gene therapies.

Over 60% of gene therapy developers are based in North AmericaOf the 110 companies developing gene therapies in the abovementioned region, 64 are start-ups, 26 are mid-sized players, while 18 are large and very large companies. Further, within this region, most of the developers are based in the US, which has emerged as a key R&D hub for advanced therapeutic products.

More than 31,000 patents have been filed / published related to gene therapies, since 2016Of these, 17% of patent applications / patents were related to gene editing therapies, while the remaining were associated with gene therapies. Leading assignees, in terms of the size of intellectual property portfolio, include (industry players) Genentech, GSK, Sangamo Therapeutics, Bayer and Novartis, (non-industry players) University of California, Massachusetts Institute of Technology, Harvard College, Stanford University and University of Pennsylvania.

USD 16.5 billion has been invested by both private and public investors, since 2014Around USD 3.3 billion was raised through venture capital financing, representing 20% of the total capital raised by industry players till June 2019. Further, there have been 28 IPOs, accounting for more than USD 2.2 billion in financing of gene therapy related initiatives. These companies have also raised significant capital in secondary offerings.

30+ mergers / acquisitions have been established between 2014 and 2019Examples of high value acquisitions reported in recent past include the acquisition of AveXis by Novartis (2018, USD 8,700 million) and Bioverativ by Sanofi (2018, USD 11,600 million).

North America and Europe are anticipated to capture over 85% of market share by 2030With a promising development pipeline and encouraging clinical results, the market is anticipated to witness an annualized growth rate of over 40% during the next decade. In addition to North America and Europe, the market in China / broader Asia Pacific region is also anticipated to grow at a relatively faster rate.

To request a sample copy / brochure of this report, please visit this link

The USD 10 billion (by 2030) financial opportunity within the gene therapy market has been analyzed across the following segments:

The report features inputs from eminent industry stakeholders, according to whom gene therapies are likely to be the most promising treatment options for genetic disorders. The report includes detailed transcripts of discussions held with the following experts:

The research covers brief profiles, featuring an overview of the therapy, current development status and clinical results. Each profile includes information on therapeutic indication, targeted gene, route of administration, special designations, mechanism of action, dosage, patent portfolio, technology portfolio, clinical trials and recent developments (if available).

For additional details, please visit https://www.rootsanalysis.com/reports/view_document/gene-therapy-market-3rd-edition-2019-2030/268.html

or email [emailprotected]

Contact:Gaurav Chaudhary+1 (415) 800 3415+44 (122) 391 1091[emailprotected]

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Gene Therapy Market is projected to be worth USD 10 Billion by 2030, growing at an annualized rate of over 40% - Scientect

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Ahwatukee Foothills parents Kendra and Dave Riley have spent most of their recent days staying at their hotel, taking their baby daughter to the nearby hospital, and making weekly trips to Amsterdam for their other daughter's medical treatment.

It's a routine they weren't expecting three months ago.

So much has changed for them since the early months of the pandemic, when the couple's middle daughter, Olivia, was diagnosed with a rare genetic disorder called metachromatic leukodystrophyf, or MLD.

Later, as Olivia began losing the ability to move and communicate, the Rileys learned their youngest daughter, Keira, also has the same disease. The Rileys flew to Milan weeks ago for an opportunity they almost missed: Gene therapy treatment that could save Keira's life.

Kendra said work flexibility helped her rearrange her family's life. She runs her own public relations company, Dawning PR, after years promoting businesses in the food and beverage industry.Prior to starting her own business, she did in-house PR for Lon's at the Hermosa Inn and helped promote Clever Koi when it first arrived onto the Phoenix dining scene.

Now, thanks to the generous donations of her community and a bit of luck as they raced against time to secure their visas in a pandemic the Rileys have a chance to start treating Keira early while moving Oliva's treatment overseas.

"I mean, every day is truly unreal to us," Kendra said in a phone call from Milan. "Yes, were handling it and moving forward and being strong for our girls. But there's not a day that goes by where I dont think, is this really happening?"

Kendra said it's been difficult to predict how each day will be because it depends on Olivia. Her middle daughter might be happy one minute, then crying the next because she's in pain or frustrated that she can't move the way she used to, Kendra said.

The support of Kendra's parents and volunteers have helped the Rileys with their day-to-day needs in Europe.

"It's mentally draining," Kendra said. "Your heart hurts when you see your child in pain."

"We didnt know what 'It takes a village' meant until we had a daughter with special needs. It truly does take a village to keep our household going," she added.

Kendra first noticed something seemed out of ordinary when Olivia appeared to have trouble walking. Olivia was about 18 months old and taking steps seemed to cause her pain. About the end of February, Kendra noticed that Olivia's irises seemed to be vibrating and an ophthalmologist suggested Olivia get an MRI.

Ahwatukee parents Kendra and Dave Riley take a stroll at their hotel with their three daughters, Eva, Olivia and Keira. The Rileys flew to Milan seeking gene therapy for Keira, who like Olivia was diagnosed with MLD.(Photo: Courtesy of Kendra Riley)

Two visits to two neurologists later, the Rileys learned Olivia had MLD.

Metachromatic leukodystrophy, or MLD, is a rare, hereditary disease that leads to premature death. It's characterized by the accumulation of fat in cells. The fat accumulation in nerve cells destroy white matter, an important tissue found in the brain and spinal cord that relays messages and affects learning functions.

Children must inherit a mutated gene from each parent to develop MLD. It's unknown how rare MLD is in the world; the incidence of MLD may range from 1 in 40,000 to 1 in 100,000, according to Cure MLD, a group that provides resources to families impacted by the disease.

The family tested their other two daughters, Eva, who is now 5, and Keira, who was 5 months old at the time. They received the results back in June: Eva is a carrier and Keira was diagnosed with MLD.

"Honestly, I feel like I had the same reaction for both," Kendra said. "An immediate feeling of being sick to your stomach, my entire body started shaking uncontrollably ... Its hard for one daughter to have this life-shortening disease. Two is too much for any parent in the world."

Olivia, who's in pain most days, has already lost the ability to talk, crawl and walk, Kendra said. In May, before transferring Olivia's treatment plan to Milan, the Rileys had begun a clinical trial in Iowa City with hopes of slowing down the disease's progression.

Through Cure MLD and the MLD Foundation, Kendra learned there was another possible option for Keira. Afairly new kind of gene therapy could potentially prolong herlife.

The catch: The therapy is only available at the San Raffaele Telethon Institute for Gene Therapy in Milan, the five-month stay including lodging and medical appointments would cost upwards of $500,000, and for the treatment to be most effective, Keira would need to start as soon as possible. With Italy banning nonessential travel from the U.S., the Rileys would need to secure passports and visas to allow them in.

FOR SUBSCRIBERS:His family inspired him to cook. Now they keep this Phoenix chef anchored during COVID

Friends, family, clients, government officials and strangers sprang into action.

A childhood friend in Yuma set up a bass fishing tournament as a fundraiser and netted around $6,000. Kendra's sister-in-law Nina Riley set up a GoFundMe that in a little more than a month, raised $243,517. Private donors pitched in the rest so the family could reach their $500,000 target.

Riley said she was amazed that even businesses that are struggling in the pandemic, such as Padre Murphy's sports bar in Glendale and Carlson Creek Vineyard, have been so supportive of her family.

"I feel like its something out of a movie, like it cant really be happening. Its not just about the donations. It's the messages Ive gotten, virtual hugs and prayers from complete strangers. All of it has restored our faith in humanity."

Through the coordinated efforts of the San Raffaele hospital, Arizona Rep. David Schweikert, the Consulate General of Italy in Los Angeles, the U.S. Postal Service and the Western Passport Center in Tucson, the Rileys were able to expedite the process of securing passports and medical visas for the entire family, plus Kendra's parents.

All of this was finalized the day before their flight to Italy, Kendra said with amazement.

Once they're back in the United States, Kendra and Dave plan to give back in some way to other families impacted by MLD. Kendra is in communication with Cure MLD to potentially work with the organization, which is described as a "global network of patient advocates and nonprofits." She and Dave are also considering starting their own foundation to support other families in situations like theirs.

"Hope is our biggest asset right now," Kendra said.

Reach the reporter at Priscilla.Totiya@azcentral.com. Follow @priscillatotiya on Twitter and Instagram.

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'Something out of a movie': How a community restored one family's 'faith in humanity' - The Arizona Republic

MINNEAPOLIS, Sept. 4, 2020 /PRNewswire/ -- Sickle Cell warrior Tahir "StayFresh" Ali of Atlanta, Ga., visits the hospital twice monthly for blood transfusions to reduce complications from sickle cell disease, and manage his potential risk of organ damage or stroke. The 35-year-old music producer and manager estimates he has spent several years of his life in the hospital undergoing treatment for sickle cell disease. Finding a perfectly matched marrow or blood stem cell donor could give StayFresh a chance to live life free of sickle cell and the many complications that the disease presents.

September is Sickle Cell Awareness Month, and Be The Match, the National Marrow Donor Program, is launching several campaigns to help people to learn more about the disease, and take action to help those searching for a matching blood stem cell or marrow donor.

StayFresh is like 100,000 African Americans who battle sickle cell disease, an often-misunderstood genetic disease that is "invisible" because unlike many illnesses, people with sickle cell disease can lead active lives. However, people with severe sickle cell can face debilitating episodes of pain crisis and life-threatening complications. StayFresh is working with Be The Match to rally more Black and African Americans to join the Be The Match Registry.

Be The Match is also hosting a three-part virtual event series to raise awareness about sickle cell disease, provide free resources for patients and families affected by sickle cell, discuss treatment options, and the need for more Black donors on the Be The Match Registry. The event series and more information can be found at: SickleCellConnect.com.

"We are grateful to have a dedicated Patient Services team that is able to produce these informative virtual events to help sickle cell patients and their caregivers learn how to access free resources and advocate for themselves as they navigate their disease," said Erica Jensen, Senior Vice President of Be The Match Member Engagement, Enrollment and Experience. "Nobody with sickle cell disease has to walk alone. Be The Match has a peer connect program, free professional counseling, nurse navigators and strong partnerships with organizations like The Sickle Cell Community Consortium and My Three Sicklers Foundation to ensure we can connect caregivers and sickle cell warriors with the resources they need to best manage this disease."

According to the Centers for Disease Control, sickle cell disease is the most common inherited disease, affecting 1 in 365 Black or African Americans and 1 in 16,300 Hispanic Americans. With sickle cell disease, oxygen-carrying red blood cells are misshapen, hard and sticky, which can result in them getting stuck in blood vessels and clog them. This can cause severe pain crisis, infections, organ damage, low blood counts, stroke and other serious health problems.

Be The Match has also launched an informative online video called "You Are My Match" to raise awareness of the need for more Black or African American donors on the Be The Match Registry to help Black patients battling sickle cell disease find a potentially life-saving match. The video features a 4-year-old girl named Ruby from Lubbock, TX, who has been hospitalized more than 24 times and had three surgeries due to her sickle cell. Ruby's doctors are recommending a blood stem cell or marrow transplant due to the severity of her condition, but she does not have a perfect match on the Be The Match Registry.

Part of the challenge Black patients like Ruby face when searching for a perfect match is low representation of Black or African Americans on the Be The Match Registry. Of the 22 million potential donors on the Be The Match Registry, just 4 percent are Black or African American. Because ethnicity plays a role in finding a match, Black patients will only find a perfect match 23% of the time, which is much lower than other ethnicities, such as White patients, who will find a match 77% of the time.

To learn how to access free resources, read stories about people cured of sickle cell disease following a marrow or stem cell transplant and to join the Be The Match Registry, visit: http://www.sicklecellconnect.com.

Virtual Event Series:Episodes will be streamed via http://www.sicklecellconnect.comwebpage and viewable on Be The Match Social Channels.

Episode 1: What is sickle cell disease?September 10thfrom 12:00 12:30 p.m. CDTWe'll chat about sickle cell disease basics with pediatric hematologist Staci Arnold, MD, hear sickle cell warrior Genesis' storyandlearn about barriers and disparities that sickle cell patients face from the Health Equity Manager at Be The Match.

Episode 2: Living with sickle cell diseaseSeptember 17th from 12:00 12:30 p.m. CDTWe'll chat with a Be The Match Patient Navigator and Social Worker about the free resources available to sickle cell disease patients and hear from the founder of My Three Sicklers Foundation.

Episode 3:The future of sickle cell diseaseSeptember24th from 12:00 1:00p.m. CDTWe'll cover transplant and gene therapy with Staci Arnold, MD and a certified genetic counselor from BeTheMatch. Hear about our policy efforts for sickle cell disease, meet sickle cell warriorDakhiyon, seehow Nia Imani Franklin, former Miss America, is advocating for more diverse donors on the Be The Match Registry, plus watch a live swabbing event!

SOURCE Be The Match

https://www.sicklecellconnect.com

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Be The Match Presents "Sickle Cell Connect" Virtual Event Series To Raise Awareness Of Treatment Options And Marrow Transplants As A Cure...