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Category Archives: Genetic Therapy

Gene Therapy Hits a Peculiar Roadblock: A Virus Shortage …

Few gene-therapy companies have the factories or expertise to make the viruses for use in clinical trials, where standards are exacting and comprehensive. Continue reading

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NCI-MATCH: Targeted Therapy Directed by Genetic Testing in …

Experimental: Subprotocol A (EGFR activating mutation) Patients with EGFR activating mutation receive afatinib orally (PO) once daily (QD) on days 1-28. Courses repeat every 28 days in the absence of disease progression or unacceptable toxicity Continue reading

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Genetic counseling – Wikipedia

Genetic counseling is the process by which the patients or relatives at risk of an inherited disorder are advised of the consequences and nature of the disorder, the probability of developing or transmitting it, and the options open to them in management and family planning. Continue reading

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Genetic Testing – Medical Clinical Policy Bulletins | Aetna

Number:0140 Aetna considers genetic testing medically necessary to establish a molecular diagnosis of an inheritable disease when all of the following are met: The member displays clinical features, or is at direct risk of inheriting the mutation in question (pre-symptomatic); and The result of the test will directly impact the treatment being delivered to the member; and Achondroplasia (FGFR3)AlbinismAlpha-1 antitrypsin deficiency (SERPINA1)Alpha thalassemia/Hb Bart hydrops fetalis syndrome/HbH disease** (HBA1/HBA2, alpha globin 1 and alpha globulin 2)Angelman syndrome (GABRA, SNRPNBardet-Biedl syndromeBeta thalassemia** (beta globin)Bloom syndrome (BLM)CADASIL (see below)Canavan disease (ASPA (aspartoacylase A))Charcot-Marie Tooth disease (PMP-22)Classical lissencephalyCongenital adrenal hyperplasia/21 hydroxylase deficiency (CYP21A2)*Congenital amegakaryocytic thrombocytopeniaCongenital central hypoventilation syndrome (PHOX2B)Congenital muscular dystrophytype 1C (MDC1C) (FKRP (Fukutin related protein))Crouzon syndrome (FGFR2, FGFR3)Cystic fibrosis (CFTR) (see below)Dentatorubral-pallidoluysian atrophyDuchenne/Becker muscular dystrophy (dystrophin)Dysferlin myopathyEhlers-Danlos syndromeEmery-Dreifuss muscular dystrophy (EDMD1, 2, and 3)Fabry diseaseFactor V Leiden mutation (F5 (Factor V))Factor XIII deficiency, congenital (F13 (Factor XIII beta globulin))Familial adenomatous polyposis coli (APC) (see below)Familial dysautonomia (IKBKAP)Familial hypocalciuric hypercalcemia (see below)Familial Mediterranean fever (MEFV)Fanconi anemia (FANCC, FANCD)Fragile X syndrome, FRAXA (FMR1) (see below)Friedreich’s ataxia (FRDA (frataxin))Galactosemia (GALT)Gaucher disease (GBA (acid beta glucosidase))Gitelman’s syndromeHemoglobin E thalassemia **Hemoglobin S and/or C **Hemophilia A/VWF (F8 ( Factor VIII))Hemophilia B (F9 (Factor IX))Hereditary amyloidosis (TTR variants)Hereditary deafness (GJB2 (Connexin-26, Connexin-32 ))Hereditary hemorrhagic telangiectasia (HHT)Hereditary hemochromatosis (HFE) (see below)Hereditary leiomyomatosis and renal cell cancer (HLRCC) syndrome (fumarate hydratase (FH) gene)Hereditary neuropathy with liability to pressure palsies (HNPP)Hereditary non-polyposis colorectal cancer (HNPCC) (MLH1, MSH2, MSH6. MSI) ( see below)Hereditary pancreatitis (PRSS1) (see below)Hereditary paraganglioma (SDHD, SDHB) Hereditary polyposis coli (APC)Hereditary spastic paraplegia 3 (SPG3A) and 4 (SPG4, SPAST) Huntington’s disease (HTT, HD(Huntington))Hypochondroplasia (FGFR3)Hypertrophic cardiomyopathy (see below)Jackson-Weiss syndrome (FGFR2)Joubert syndromeKallmann syndrome (FGFR1)Kennedy disease (SBMA)Leber hereditary optic neuropathy (LHON)Leigh Syndrome and NARP (neurogenic muscle weakness, ataxia, and retinitis pigmentosa) Long QT syndrome (see below)Limb girdle muscular dystrophy (LGMD1, LGMD2) (FKRP (Fukutin related protein))Malignant hyperthermia (RYR1)Maple syrup urine disease (branched-chain keto acid dehydrogenase E1)Marfans syndrome (TGFBR1, TGFBR2)McArdle’s diseaseMedium chain acyl coA dehydrogenase deficiency (ACADM)Medullary thyroid carcinomaMELAS (mitochondrial encephalomyopathy with lactic acidosis and stroke-like episodes) (MTTL1, tRNAleu)Meckel-Gruber syndromeMucolipidosis type IV (MCOLN1, mucolipin 1)Mucopolysaccharidoses type 1 (MPS-1)Muenke syndrome (FGFR3)Multiple endocrine neoplasia type 1Muscle-Eye-Brain disease (POMGNT1)MYH-associated polyposis (MYH) (see below)Myoclonic epilepsy (MERRF) (MTTK (tRNAlys))Myotonic dystrophy (DMPK, ZNF-9)Neimann-Pick disease, type A(SMPD1, sphingomyelin phosphodiesterase)Nephrotic syndrome, congenital (NPHS1, NPHS2)Neurofibromatosis type 1 (NF1, neurofibromin)Neurofibromatosis type 2 (Merlin)Neutropenia, congenital cyclicNephronophthisisPhenylketonuria (PAH)Pfeiffer syndrome (FGFR1)Prader-Willi-Angelman syndrome (SNRPN, GABRA5, NIPA1, UBE3A, ANCR, GABRA )Primary dystonia (TOR1A (DYT1))Prothrombin (F2 (Factor II,20210G> A mutation))Pyruvate kinase deficiency (PKD)Retinoblastoma (Rh)Rett syndrome (FOXG1, MECP2)Saethre-Chotzen syndrome (TWIST, FGFR2)SHOX-related short stature (see below)Smith-Lemli-Opitz syndromeSpinal muscular atrophy (SMN1, SMN2)Spinocerebellar ataxia (SCA types 1, 2, 3 (MJD), 6 (CACNA1A), 7, 8, 10, 17 and DRPLA)Tay-Sachs disease (HEXA (hexosaminidase A))Thanatophoric dysplasia (FGFR3)Von Gierke disease (G6PC, Glycogen storage disease, Type 1a)Von Hippel-Lindau syndrome (VHL)Walker-Warburg syndrome (POMGNT1)22q11 deletion syndromes (DCGR (CATCH-22)) * Medically necessary if results of the adrenocortical profile following cosyntropin stimulation test are equivocal or for purposes of genetic counseling. Continue reading

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A bioethicist’s take on child cancer treatment that uses gene therapy – The Tidings

A genetic modification therapy designed for treating pediatric leukemia has drawn both praise and caution from a Catholic bioethicist, after recently being approved by the Food and Drug Administration. While a promising use of gene modification technology, the treatment still has potentially deadly side effects – which could make the risks outweigh the benefits, says Fr Continue reading

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Pioneering cancer gene therapy gets green light and $475,000 … – Salon

This article originally appeared on Kaiser Health News The countrys first approved gene therapy approved Wednesday to fight leukemia that resists standard therapies will cost $475,000 for a one-time treatment, its manufacturer announced. Switzerland-based Novartis, which makes the innovative therapy, announced that the drug will cost nothing if patients fail to benefit in the first month. TheFood and Drug Administrationapproved the therapy, called Kymriah, in children and young adults with acute lymphoblastic leukemia whose disease has come back in spite of previous treatments Continue reading

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