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Poxel Announces the Formation of its Scientific Advisory Board for Rare Metabolic Diseases – Yahoo Finance

LYON, France, December 06, 2021--(BUSINESS WIRE)--POXEL SA (Euronext POXEL - FR0012432516), a clinical stage biopharmaceutical company developing innovative treatments for chronic diseases with metabolic pathophysiology, including non-alcoholic steatohepatitis (NASH) and rare disorders, today announced the formation of its Scientific Advisory Board (SAB) for rare metabolic diseases. The new SAB will initially focus on supporting Poxel's X-linked adrenoleukodystrophy (ALD) program.

"I am delighted to welcome this distinguished group of scientific thought leaders to Poxels SAB for rare metabolic diseases. These therapeutic indications, including ALD, are areas of unsurpassed unmet medical need where treatments are not available or very limited. We are grateful for their informed counsel and deep knowledge and experience that will help shape Poxels discovery and clinical-stage programs and further advance our mission to develop therapies for rare metabolic diseases. We look forward to continuing to collaborate with this accomplished group of advisors as we expand our clinical programs, and initiate Phase 2a studies for ALD in early 2022, with both PXL065, a novel deuterium modified thiazolidinedione and PXL770, a first-in-class direct AMPK activator," said David E. Moller, Poxel's Chief Scientific Officer.

Poxel recently hosted its first SAB meeting on rare diseases, which led to highly productive discussions regarding the potential of its compounds and the future of its programs in ALD, including the design of its two proof-of-concept (POC) Phase 2a studies which the Company intends to initiate early 2022.

The members of the Rare Metabolic Diseases Scientific Advisory Board are the following:

Prof. Florian Eichler, MD, is an expert in inherited diseases with metabolic pathophysiology that affect the nervous system. Dr. Eichler received training in pediatric neurology and neurogenetics at Harvard and Johns Hopkins. He is currently the director of the Center for Rare Neurologic Disorders and the director of the Leukodystrophy Service at Massachusetts General Hospital (MGH), Harvard Medical School, US. His research focus is on the genetics of peroxisomal disorders, lipid metabolism, and gene therapy for neurodegenerative diseases. Dr. Eichler is also President of ALD Connect.

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Prof. Marc Engelen, MD, PhD, is a clinical researcher, a specialist in pediatric neurology, gastroenterology and endocrinology, and an expert in peroxisomal disorders and leukodystrophies at Amsterdam University Medical Centers, Netherlands. Dr. Engelen received his MD and PhD degrees at the University of Amsterdam. Along with Stephan Kemp and other members of his team, Dr. Engelen's research efforts have unveiled novel approaches to detecting, diagnosing and monitoring neurometabolic diseases including ALD.

Prof. S. Ali Fatemi, MD, is a physician-scientist leader in pediatric neurology and neurometabolic diseases. Dr. Fatemi was trained at the Medical University of Vienna, Austria and at Kennedy Krieger Institute, Johns Hopkins, US. He founded the Moser Center for Leukodystrophies and is now Chief Medical Officer at the Kennedy Krieger Institute, Baltimore, US. His research is focused on basic and translational studies pertaining to the pathophysiology of ALD.

Prof. Stephan Kemp, PhD, is a translational research expert in genetic neurometabolic diseases. He was trained at Johns Hopkins University, Kennedy Krieger Institute, and at the University of Amsterdam, where he is a longstanding faculty member. His research focuses on lipid metabolism and neurotoxicity and he has published many seminal papers on the pathobiology of ALD. He also leads efforts focused on newborn screening for rare metabolic disorders in the Netherlands.

Prof. Fanny Mochel, MD, PhD, is an expert in inborn errors of metabolism. She received training in genetics and neuroscience at University Pierre and Marie Curie in Paris and she is a faculty member in genetics at this university. Dr. Mochel also leads the French reference Center on Neurometabolic diseases and is co-chair of the French society for inborn of errors of metabolism and a council member of the Society for the Study of Inborn Errors of Metabolism. Her research efforts include characterization and treatment of brain energy deficiencies in neurometabolic disease, the identification of novel biomarkers, metabolomics and in vivo metabolic imaging, as well as therapeutic approaches targeting the Krebs cycle.

Dr. Jaspreet Singh, PhD, is a neuroscientist researcher focusing on ALD pathophysiology, neuroinflammation, biomarker development and testing novel therapeutic options. He was trained in India and at the Medical University of South Carolina, US. He is currently a faculty member in the Department of Neurology at the Henry Ford Health System in Detroit, US.

Prof. Keith Van Haren, MD, is a pediatric neurologist and an expert in ALD. He received medical and specialty training at the University of Rochester Medical School, Harvard Medical School, and Stanford University, US. Dr. Van Haren is a faculty member in Neurology and Neurological Sciences at Stanford. He cares for patients including many with ALD and also leads a laboratory focusing on the study of single-gene mutations and attendant molecular mechanisms leading to neuroinflammation in humans.

About ALDX-linked adrenoleukodystrophy (ALD) is an orphan neurometabolic disease caused by mutations in the ABCD1 gene which encodes for a key protein that is required for metabolism of very long chain fatty acids (VLCFA) by peroxisomes (cellular organelles). ALD is the most common leukodystrophy with a prevalence similar to hemophilia up to 1/10,000 individuals in the general population have ALD [https://rarediseases.org]. Forms of this disease include cerebral ALD (C-ALD) and adrenomyeloneuropathy (AMN) which is the most common form typically occurring in adolescence through adulthood. AMN is characterized by chronic and progressive distal axonopathy involving the long tracts of the spinal cord and to a lesser extent the peripheral nerves resulting in progressive stiffness and weakness in the legs, impaired gait and balance, incontinence, and loss of sensation. All men are affected, and many women also present with features of AMN with a later onset. C-ALD is characterized by inflammatory demyelination of cells in the brain and typically afflicts children, but many men with AMN may also develop cerebral disease; these white matter brain lesions lead to severe neurologic deficits and death. There are no approved medicines for ALD (other than glucocorticoid supplements for associated adrenal insufficiency). C-ALD when first detected in early childhood, can be treated with hematopoietic stem cell transplantation. HSCT is currently limited to early stage of C-ALD and this procedure is at risk of severe adverse reactions.

About Poxel SA

Poxel is a clinical stage biopharmaceutical company developing innovative treatments for chronic serious diseases with metabolic pathophysiology, including non-alcoholic steatohepatitis (NASH) and rare disorders. Poxel has clinical and earlier-stage programs from its adenosine monophosphate-activated protein kinase (AMPK) activator and deuterated TZD platforms targeting chronic and rare metabolic diseases. For the treatment of NASH, PXL065 (deuterium-stabilized R-pioglitazone) is in a streamlined Phase 2 trial (DESTINY-1). PXL770, a first-in-class direct AMPK activator, has successfully completed a Phase 2a proof-of-concept trial for the treatment of NASH, which met its objectives. For the rare inherited metabolic disorder, adrenoleukodystrophy (ALD), the company intends to initiate Phase 2a proof of concept studies with PXL065 and PXL770 in patients with adrenomyeloneuropathy (AMN). TWYMEEG (Imeglimin), Poxels first-in-class lead product that targets mitochondrial dysfunction, has been approved and launched for the treatment of type 2 diabetes in Japan. Poxel expects to receive sales-based payments and royalties from Sumitomo Dainippon Pharma. Poxel has a strategic partnership with Sumitomo Dainippon Pharma for Imeglimin in Japan, China, South Korea, Taiwan and nine other Southeast Asian countries. The Company intends to generate further growth through strategic partnerships and pipeline development. Listed on Euronext Paris, Poxel is headquartered in Lyon, France, and has subsidiaries in Boston, MA, and Tokyo, Japan.

For more information, please visit: http://www.poxelpharma.com

All statements other than statements of historical fact included in this press release about future events are subject to (i) change without notice and (ii) factors beyond the Companys control. These statements may include, without limitation, any statements preceded by, followed by or including words such as "target," "believe," "expect," "aim," "intend," "may," "anticipate," "estimate," "plan," "project," "will," "can have," "likely," "should," "would," "could" and other words and terms of similar meaning or the negative thereof. Forward-looking statements are subject to inherent risks and uncertainties beyond the Companys control that could cause the Companys actual results or performance to be materially different from the expected results or performance expressed or implied by such forward-looking statements.

View source version on businesswire.com: https://www.businesswire.com/news/home/20211205005012/en/

Contacts

Poxel SA Catherine DavidInvestor Relations & Communication Managercatherine.david@poxelpharma.com +33 7 64 57 61 78

Aurlie BozzaInvestor Relations & Communication Directoraurelie.bozza@poxelpharma.com +33 6 99 81 08 36

Elizabeth WooSenior Vice President, Investor Relations & Communicationelizabeth.woo@poxelpharma.com

Investor relations / Media NewCapEmmanuel Huynh or Arthur Rouillpoxel@newcap.eu +33 1 44 71 94 94

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Poxel Announces the Formation of its Scientific Advisory Board for Rare Metabolic Diseases - Yahoo Finance

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Experts stark warning over wave of neurological illness to follow Covid infections – Express

The chronic and short-term complications of coronavirus have remained in sharp focus throughout the pandemic. But as research advances, its becoming increasingly apparent that certain patients find their brains continue to bear out the scars of their COVID-19 battles. Data released last year raised the alarm over the cases of three young COVID-19 patients who developed Parkinson's within weeks of contracting the virus. An expert has now warned that many more could be at the perils of the neurodegenerative condition in the coming years.

Kevin Barnham from Florey Institute of Neuroscience and Mental Health warned that a wave of neurological illness is set to follow the pandemic.

Parkinsons disease is a complex illness, but one of the causes is inflammation, and the virus helps to drive that inflammation," explained Miss Barnham.

Once the inflammation gets into the brain, it starts a cascade of events which can ultimately lead to Parkinsons disease.

Evidence is already suggesting the triggers for Parkinsons disease are there with this virus. I believe the risk is real.

READ MORE: Parkinson's: Exactly how much water you should drink to avoid Parkinson's symptoms

We cant put a number on it, but with 30 million people worldwide affected by this virus, even a small shift in the risk of getting Parkinsons would lead to many more people getting diagnosed.

We know COVID-19 has short-term effects, but we are releasing more about the potential long-term effects.

Data published in November of 2020 were the first to raise the alarm over potential neurological implications of infection with COVID-19.

The data drew on three separate case reports on relatively young COVID-19 patients who developed Parkinsons within two to five weeks of contracting the disease.

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The lead author of the article, Patrik Brundin, warned at the time: If this link is real, we might be in for an epidemic of Parkinsons disease in the future."

The three patients, aged 35, 45 and 58-years-old respectively, all incurred a severe respiratory infection from Covid, which led to their hospitalisation.

Brain imaging later revealed classic signs of Parkinsons disease in all three patients.

These cases of acute Parkinsons in patients with COVID-19 are truly remarkable," noted Mr Brundin.

They occurred in relatively young people - much younger than the average age of developing Parkinsons - and none had a family history of early signs of Parkinsons prodrome. That is quite a stunning observation.

Parkinsons is normally a very slowly developing disease, but in these cases, something happened quickly.

The doctor suggested the virus might make patients susceptible to Parkinsons by plaguing them with neurological symptoms after infection.

These typically include brain fog and depressions, which are consistent with damage to the brain and could lead to Parkinsons.

Parkinson's disease is characterised by a gradual shortfall of dopamine in the brain, the hormone responsible for movement in the body.

This causes problems with body movement, including involuntary tremors and rigidity, both of which can severely compromise quality of life.

In light of the alarming findings, researchers have suggested patients undergo early tests to pick up symptoms.

Parkinsons patients can experience loss of smell up to a decade before the onset of symptoms, so a smell-test screening could open up the window opportunity for early medical intervention, explained doctor Lyndsea Collins-Praino, Head of cognition ageing laboratory at the University of Adelaide.

Doctor Collins-Praino, added: The earlier we can detect [the damage] the better our chances of really effective and meaningful therapeutics for individuals.

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Experts stark warning over wave of neurological illness to follow Covid infections - Express

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Minimally Invasive Neurosurgical Systems Industry to 2030 Rising cases of neurological disorders and growing demand for efficient minimally invasive…

The global minimally invasive neurosurgical systems market size was US$ 467 million in 2021. The global minimally invasive neurosurgical systems market is forecast to grow to US$1,162 million by 2030, growing at a compound annual growth rate (CAGR) of 10.5% during the forecast period from 2022 to 2030.

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The use of minimally invasive neurosurgical systems can be helpful in many ways. It helps surgeons have better visualization than the traditional methods. Moreover, it allows a small incision of the skull during the surgical process.

Factors Influencing

Geographic Overview

North America is forecast to dominate the global minimally invasive neurosurgery system market due to the rapidly rising cases of neurological disorders. Moreover, the increasing penetration of advanced technology and high healthcare expenditure will benefit the global market.

In addition, the rapidly developing healthcare sector and increasing awareness about minimally invasive neurosurgical treatments among the population are forecast to benefit the regional market.

COVID-19 Impact Analysis

Due to the COVID-19 pandemic, the global minimally invasive neurosurgical systems market witnessed an abrupt decline. The authorities and healthcare bodies all around the world were concentrating on curbing the effect of the virus and saving the lives of the people. As a result of this, neurological surgeries were either postponed or canceled for a short period of time. Moreover, patients also stopped visiting hospitals and clinics to prevent themselves from the effect of this deadly virus. Thus, the global minimally invasive neurosurgical systems market witnessed a significant decline.

The global minimally invasive neurosurgical systems market also slowed down due to the unavailability of raw materials, which created various challenges for the market players.

Competitors in the Market

Market Segmentation

The global minimally invasive neurosurgical systems market is segmented on the basis of Product, Surgery Type, and Region.

Insight by Product Type

Insight by Surgery Type

Insight by Region

The global minimally invasive neurosurgical systems market size was US$ 467 million in 2021. The global minimally invasive neurosurgical systems market is forecast to grow to US$1,162 million by 2030, growing at a compound annual growth rate (CAGR) of 10.5% during the forecast period from 2022 to 2030.

The use of minimally invasive neurosurgical systems can be helpful in many ways. It helps surgeons have better visualization than the traditional methods. Moreover, it allows a small incision of the skull during the surgical process.

Factors Influencing

Geographic Overview

North America is forecast to dominate the global minimally invasive neurosurgery system market due to the rapidly rising cases of neurological disorders. Moreover, the increasing penetration of advanced technology and high healthcare expenditure will benefit the global market.

In addition, the rapidly developing healthcare sector and increasing awareness about minimally invasive neurosurgical treatments among the population are forecast to benefit the regional market.

COVID-19 Impact Analysis

Due to the COVID-19 pandemic, the global minimally invasive neurosurgical systems market witnessed an abrupt decline. The authorities and healthcare bodies all around the world were concentrating on curbing the effect of the virus and saving the lives of the people. As a result of this, neurological surgeries were either postponed or canceled for a short period of time. Moreover, patients also stopped visiting hospitals and clinics to prevent themselves from the effect of this deadly virus. Thus, the global minimally invasive neurosurgical systems market witnessed a significant decline.

The global minimally invasive neurosurgical systems market also slowed down due to the unavailability of raw materials, which created various challenges for the market players.

Competitors in the Market

Market Segmentation

The global minimally invasive neurosurgical systems market is segmented on the basis of Product, Surgery Type, and Region.

Insight by Product Type

Insight by Surgery Type

Insight by Region

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Minimally Invasive Neurosurgical Systems Industry to 2030 Rising cases of neurological disorders and growing demand for efficient minimally invasive...

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Saving patients an unnecessary procedure – EurekAlert

A new study from The Neuro (Montreal Neurological Institute-Hospital) and eight collaborating international epilepsy centers has developed a simple web-based application clinicians can use to predict which patients will not benefit from an invasive diagnostic work-up, preventing unnecessary, invasive procedures, saving time for patients and the clinical team, and freeing up overburdened health resources.

Surgery is the only option to cure seizures inpatients with drug-resistant epilepsy. In evaluating patients for surgery, clinicians have to pinpoint what brain region the seizures are coming from. The way to do this in patients with more complex epilepsy is through stereo-electroencephalography (SEEG), a technique by which a surgeon implants electrodes into the brain to find the source of the seizures.

SEEG is an invasive procedure that requires a one-to-two-week hospital stay and comes with risk of infection, bleeding and stroke in 0.5-2 per cent of procedures. Furthermore, in up to 42 per cent of case series, SEEG does not result in an epilepsy surgical intervention as no focal epileptic focus can be identified. To reduce the amount of people undergoing an unnecessary SEEG, researchers studied epilepsy patients to determine if a series of non-invasive tests could predict which patients would not benefit from SEEG.

The team followed 128 patients at The Neuro who had SEEG, analyzing their demographic, clinical, electroencephalography, neuroimaging, and neuropsychological data. They developed a regression model based on different modalities, called the 5-SENSE-score, that differentiated patients whose SEEG identified a defined seizure source from those that did not. They then validated this score on a larger cohort of 207 patients from nine different tertiary epilepsy centres, finding that it reliably predicted the patients in whom SEEG was unable to identify a focal seizure onset zone.

Many epilepsy centres face the challenging decision of whether a patient should undergo implantation for identifying a focal-seizure onset zone, says Dr. Birgit Frauscher, neurologist at The Neuro and the studys senior author. The 5-SENSE-score provides an easily applicable tool to guide clinicians in predicting patients where SEEG is unlikely to identify a focal seizure onset zone. Patients unlikely to benefit from this invasive and resource-intensive procedure can then be identified earlier, avoiding unnecessary burden on patients and overutilization of hospital resources.

This study, published in the Journal of the American Medical Association on Dec. 6, 2021, was funded by the Montreal Neurological Institute, the Fonds de Recherche du Qubec Sant, and the Austrian Chapter of the International League against Epilepsy.

The Neuro

The Neuro The Montreal Neurological Institute-Hospital is a bilingual, world-leading destination for brain research and advanced patient care. Since its founding in 1934 by renowned neurosurgeon Dr. Wilder Penfield, The Neuro has grown to be the largest specialized neuroscience research and clinical center in Canada, and one of the largest in the world. The seamless integration of research, patient care, and training of the worlds top minds make The Neuro uniquely positioned to have a significant impact on the understanding and treatment of nervous system disorders. In 2016, The Neuro became the first institute in the world to fully embrace the Open Science philosophy, creating the Tanenbaum Open Science Institute. The Montreal Neurological Institute is a McGill University research and teaching institute. The Montreal Neurological Hospital is part of the Neuroscience Mission of the McGill University Health Centre. For more information, please visitwww.theneuro.ca

Data/statistical analysis

People

Development and Validation of the 5-SENSE Score to Predict Focality of the Seizure-Onset Zone as Assessed by Stereoelectroencephalography

6-Dec-2021

Disclaimer: AAAS and EurekAlert! are not responsible for the accuracy of news releases posted to EurekAlert! by contributing institutions or for the use of any information through the EurekAlert system.

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Neurologist at centre of NI’s biggest ever patient recall Dr Michael Watt will contest decision to appeal his voluntary removal from medical register…

A neurologist at the centre of Northern Ireland's biggest ever patient recall will contest a decision to appeal his voluntary removal from the medical register, the High Court heard today.

ounsel for Dr Michael Watt confirmed his intention to resist the surprise intervention by an oversight body.

Lawyers representing former patients welcomed the step taken by the Professional Standards Authority (PSA), but stressed they must not be shut out from the legal process.

In October the Medical Practitioners Tribunal Service (MPTS) granted Dr Watt's application for voluntary erasure from the register.

The regulator's controversial decision means the former Belfast Trust consultant will not face a public hearing into concerns about his work.

His inability to engage with a fitness to practice assessment and the potential risk of suicide were cited among the reasons.

Some of those Dr Watt treated are seeking a judicial review of the lawfulness of the decision.

Danielle O'Neill, 39, claims there was no jurisdiction for the move which breaches her human rights.

Belfast man Michael McHugh, 51, also alleges it was an unjust step, denying public scrutiny of the neurologist's work.

But it has now emerged that the PSA, which oversees regulators, has lodged a High Court appeal against the MPTS decision to allow voluntary removal.

A judge questioned whether the judicial review challenges should be put on hold pending the outcome of the separate legal action.

He was told that the neurologist intends to oppose the PSA's challenge.

David Dunlop QC confirmed: "It will come as no surprise that Dr Watt will be contesting the appeal, on the grounds that it seeks to challenge the basis on which the voluntary erasure application was approved."

During the hearing counsel representing Ms O'Neill, Dessie Hutton QC, reiterated claims that the MPTS decision was a nullity.

"If that argument is correct, there was never any basis for the decision, and there is no basis for the appeal," he submitted.

Adjourning proceedings, Mr Justice Colton stressed all of the cases should be reviewed again as soon as possible.

Outside court Ms O'Neill insisted that her legal action represents an opportunity for patients' to be heard.

She added: "I find it galling that Dr Watt, who should have a back seat in these proceedings, after all we have suffered, is now seeking to dictate how our cases will be heard."

Her solicitor claimed the voluntary removal decision had closed off the only remaining public hearing into alleged clinical failings which have caused "harm and immeasurable distress".

Claire McKeegan of Phoenix Law said: "Our clients (in the) Neurology Recall Support Group, who have been injured and traumatised, welcome the intervention of the Professional Standards Authority but wish to have a voice and participate in the court process." end

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Shift towards Orphan Drugs is Anticipated to Drive Demand Growth in Rare Neurological Disease Treatm – PharmiWeb.com

250 Pages Rare Neurological Disease Treatment Market Survey by Fact MR, A Leading Business and Competitive Intelligence Provider

According to Fact MRs recent market research,sales of Rare Neurological Disease Treatmentto swell at propelled CAGR through2031, as investment towards expansion of healthcare infrastructure continues rising. The goal of the survey is uncover growth opportunities, key trends, growth drivers, and challenges. It also provides recommendations to assist businesses in preparing for unforeseen challenges.

Focus on improving overall patient care will remain a chief growth driver. Besides this, Rare Neurological Disease Treatment market will continue gaining from increasing willingness among patients and families to spend on advanced healthcare.

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Healthcare organizations around the world are deploying digital tools to accommodating changing consumer preferences. They are pressing on solving the long-standing challenge pertaining to care model innovation.

Considering this, the focus on making healthcare affordable and more transparent will increase. Adoption of care model innovation in healthcare will have a profound impact on the Rare Neurological Disease Treatment market.

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This newly published and insightful report sheds light on Market Insights of Rare Neurological Disease Treatment, key dynamics, their impact on the overall value chain from suppliers to end-users and Growth of Rare Neurological Disease Treatment Market.

The global rare neurological disease treatment market is segmented on the basis of drug class, route of administration, disease indication, distribution channel, and region.

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This taxonomy and the detailed TOC prepared are confidential and intended exclusively for the individual or entity with whom it is being shared.

Reading, disseminating, distributing, or copying this to any party other than addressee(s) is unauthorized and prohibited.

Country-specific assessment on demand for rare neurological disease treatment has been provided for each regional market, along with the market size valuation and forecast price point assessment, price index and impact analysis of key regional and country-wise dynamics, which were obtained through quotes from numerous rare neurological disease treatment manufacturers, experts, and suppliers.

Y-o-Y growth projections have also been offered on all regional markets incorporated in the report. Moreover, future trends, growth prospects, and other possibilities related to the market have been answered in the report.

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The report provides detailed competitive and company profiles of key participants operating in the global rare neurological disease treatment market.

Some of the key competitors operating in the rare neurological disease treatment market are Novartis AG, Pfizer, Inc., Johnson & Johnson Services, Inc. Bayer AG, GlaxoSmithKline Plc, Sanofi S.A., Allergan Plc, Merck & Co., Inc., and others.

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Shift towards Orphan Drugs is Anticipated to Drive Demand Growth in Rare Neurological Disease Treatm - PharmiWeb.com

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