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Total 12 results found since Jan 2013.
Astellas and Kate Therapeutics Announce Exclusive License Agreement for KT430
KT430 is a preclinical, next-generation investigational gene therapy to treat X-linked myotubular myopathy (XLMTM)
TOKYO and SAN DIEGO, June 8, 2023 -- (Healthcare Sales & Marketing Network) -- Astellas Pharma Inc. (TSE: 4503, President and CEO: Naoki ... Biopharmaceuticals, Regenerative Medicine, Licensing Astellas Pharma, Kate Therapeutics, X-linked myotubular myopathy, gene therapy
Source: HSMN NewsFeed - June 8, 2023 Category: Pharmaceuticals Source Type: news
The new horizons for treatment of Late-Onset Pompe Disease (LOPD)
Rev Neurol (Paris). 2023 Jan 4:S0035-3787(22)00847-5. doi: 10.1016/j.neurol.2022.12.004. Online ahead of print.ABSTRACTLate-onset Pompe disease (LOPD) is a genetic myopathy causing skeletal muscle weakness and severe respiratory impairment, due to the deficiency of the lysosomal enzyme acid alpha-glucosidase (GAA) leading to lysosomal glycogen accumulation along with other complex pathophysiological processes. A major step for treatment of Pompe disease was reached in 2006 with the marketing of alglucosidase alfa, a first enzyme replacement therapy (ERT) that showed a significant motor and respiratory benefit. However, eff...
Source: Revue Neurologique - January 7, 2023 Category: Neurology Authors: C Gu émy P Lafor êt Source Type: research
Collagen related muscle diseases
Mutations in the Col6 genes (COL6A1, COL6A2 and COL6A3) cause a clinically and genetically heterogeneous group of rare diseases (Bethlem myopathy, BM; Ullrich congenital muscular dystrophy, UCMD; intermediate phenotypes), which are collectively known as the COL6-related muscle diseases (COL6-MDs). The Global Registry for COL6-related muscle disease allows secure capture and storage of data from individuals affected with a COL6-MD and from the medical professionals in charge of their care. The primary objectives of the registry are to: contribute to trial readiness of COL6-MDs, allowing identification of genetically well ch...
Source: Neuromuscular Disorders - September 19, 2021 Category: Neurology Authors: L. Imber, A. Blain, V. Straub Source Type: research
Registries and care of nmd
The GNE myopathy registry is an international, patient reported, disease specific database which provides the neuromuscular community with a valuable, flexible and sustainable resource of disease specific information. The registry can be used to support planning and recruitment for clinical trials, and to capture real-world patient data (including data that may contribute to post marketing surveillance and standards of care). The GNE registry is managed by the John Walton muscular dystrophy research centre (JWMDRC, Newcastle University, UK) and supervised by an international steering committee.
Source: Neuromuscular Disorders - September 19, 2021 Category: Neurology Authors: L. Imber, V. Straub Source Type: research
Audentes Therapeutics Board of Directors Appoints Co-Founder and Chief Executive Officer Matthew R. Patterson as Chairman
Louis G. Lange, M.D., Ph.D. appointed as Lead Independent Director
SAN FRANCISCO, Nov. 5, 2018 -- (Healthcare Sales & Marketing Network) -- Audentes Therapeutics, Inc. (Nasdaq: BOLD), a biotechnology company focused on developing and commercializing in... Biopharmaceuticals, Personnel Audentes Therapeutics, Myotubular Myopathy, Crigler-Najjar syndrome
Source: HSMN NewsFeed - November 5, 2018 Category: Pharmaceuticals Source Type: news
Audentes Announces Priority Medicines (PRIME) Designation Granted by the European Medicines Agency to AT132 for the treatment of X-Linked Myotubular Myopathy
SAN FRANCISCO, June 5, 2018 -- (Healthcare Sales & Marketing Network) -- Audentes Therapeutics, Inc. (Nasdaq: BOLD), a biotechnology company focused on developing and commercializing innovative gene therapy products for patients living with serious, life-... Biopharmaceuticals, Regulatory Audentes Therapeutics, gene therapy, Myotubular Myopathy
Source: HSMN NewsFeed - June 5, 2018 Category: Pharmaceuticals Source Type: news
Audentes Therapeutics Appoints Mark A. Goldberg, M.D. to its Board of Directors
SAN FRANCISCO, Dec. 13, 2017 -- (Healthcare Sales & Marketing Network) -- Audentes Therapeutics, Inc. (Nasdaq: BOLD), a biotechnology company focused on developing and commercializing gene therapy products for patients living with serious, life-threatenin... Biopharmaceuticals, Personnel Audentes Therapeutics, gene therapy, X-Linked Myotubular Myopathy
Source: HSMN NewsFeed - December 13, 2017 Category: Pharmaceuticals Source Type: news
Stealth BioTherapeutics Granted Fast Track Designation for the Treatment of Barth Syndrome
BOSTON, Nov. 27, 2017 -- (Healthcare Sales & Marketing Network) -- Stealth BioTherapeutics (Stealth), a clinical-stage biopharmaceutical company developing therapeutics to treat mitochondrial dysfunction, today announced that the U.S. Food and Drug Admini... Biopharmaceuticals, FDA Stealth BioTherapeutics, elamipretide, Barth Syndrome, mitochondrial myopathy
Source: HSMN NewsFeed - November 27, 2017 Category: Pharmaceuticals Source Type: news
Audentes Therapeutics Announces Publication of Data from RECENSUS, a Retrospective Medical Record Review of Patients With X-Linked Myotubular Myopathy
SAN FRANCISCO, Nov. 21, 2017 -- (Healthcare Sales & Marketing Network) -- Audentes Therapeutics, Inc. (Nasdaq: BOLD), a biotechnology company focused on developing and commercializing gene therapy products for patients living with serious, life-threatenin... Biopharmaceuticals Audentes Therapeutics, X-Linked Myotubular Myopathy, RECENSUS Study
Source: HSMN NewsFeed - November 21, 2017 Category: Pharmaceuticals Source Type: news
Ionis Licenses Novel Antisense Drug for the Treatment of Centronuclear Myopathy to Dynacure
Ionis earns $5 million license fee from Dynacure
CARLSBAD, Calif. and STRASBOURG, France, Nov. 9, 2017 -- (Healthcare Sales & Marketing Network) -- Ionis Pharmaceuticals, Inc. (NASDAQ: IONS) and Dynacure announced today that Dynacure has licensed IONIS... Biopharmaceuticals, Licensing Ionis Pharmaceuticals, Dynacure, centronuclear myopathy
Source: HSMN NewsFeed - November 9, 2017 Category: Pharmaceuticals Source Type: news
Digitalization and Pharma, Opportunity or Irrelevance?
The pace of healthcare evolution and change is not slowing. This offers opportunities to pharma companies to positively support and ease the pain of transition and change and digital technology provides a means of fulfilling these opportunities.
Cost control and outcome-based care
Government and payors are creating and experiencing increasing pressure to manage costs, including the purchase and use of medicine. Being accepted and retained on formularies may be influenced by more cost-effective delivery and compliance processes, supported digitally. For example, sensors and personal dispensing devices can collect and analys...
Source: EyeForPharma - May 31, 2016 Category: Pharmaceuticals Authors: Pamela Walker Source Type: news
