Patient in Sarepta Gene Therapy Trial Develops Serious Illness Patient in Sarepta Gene Therapy Trial Develops Serious Illness

Shares of Sarepta Therapeutics Inc plunged 12% after a notice from the U.S. health agency showed a patient in an ongoing study of the company's gene therapy for Duchenne muscular dystrophy (DMD) was reported to have developed a serious illness.Reuters Health Information
Source: Medscape Today Headlines - Category: Consumer Health News Tags: Neurology & Neurosurgery News Source Type: news

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Duchenne Muscular Dystrophy (DMD) is the most common muscle disease in children, and there are no effective therapies for DMD or Becker Muscular Dystrophy (BMD). Currently, targeted gene therapy treatments hav...
Source: BMC Medical Genetics - Category: Genetics & Stem Cells Authors: Tags: Research article Source Type: research
Sarepta Therapeutics Inc said on Thursday it was informed that an adverse event report was erroneously submitted to the U.S. health regulator regarding an ongoing study of the company's gene therapy for Duchenne muscular dystrophy (DMD).
Source: Reuters: Health - Category: Consumer Health News Tags: healthNews Source Type: news
Sarepta Therapeutics stock dropped as much as 13 percent Thursday after news emerged that a child participating in a trial of the company's experimental gene therapy for Duchenne muscular dystrophy experienced a serious health complication. Sarepta (Nasdaq: SRPT) has been testing a treatment for the rare disease that would insert a functioning gene into patients with the disease, who have a non-working copy of the gene. The Cambridge biotech's stock quickly dropped Thursday when it was reported…
Source: bizjournals.com Health Care:Biotechnology headlines - Category: Biotechnology Authors: Source Type: news
Shares of Sarepta Therapeutics Inc plunged 12% after a notice from the U.S. health agency showed a patient in an ongoing study of the company's gene therapy for Duchenne muscular dystrophy (DMD) was reported to have developed a serious illness.
Source: Reuters: Health - Category: Consumer Health News Tags: healthNews Source Type: news
Conclusion Our data indicated that the MLPA plus NGS can be a comprehensive and effective tool for precision diagnosis and potential treatment of MD, and is particularly necessary for the patients at very young age with only two clinical indicators (persistent hyperCKemia and typical myopathy performance on electromyogram) but no definite clinical manifestations.
Source: Frontiers in Pharmacology - Category: Drugs & Pharmacology Source Type: research
CONCLUSION: Gene therapies have the potential to significantly influence the course of neuromuscular diseases. First positive intermediate results have been published and the first treatment has recently been approved in the USA. Long-term data on sustained effects and toxicity of gene therapies are not yet available. These novel treatment options will present new challenges for the healthcare systems concerning diagnosis, treatment and reimbursement. PMID: 31286145 [PubMed - as supplied by publisher]
Source: Der Nervenarzt - Category: Neurology Authors: Tags: Nervenarzt Source Type: research
Abstract Introduction Genetic neuromuscular diseases (NMDs) constitute a heterogeneous group of rare conditions, including some of the most disabling conditions in childhood. Over the last decade, early diagnosis, multidisciplinary care and anticipatory treatment strategies have improved survival and quality of life of several conditions. Recently, advanced technologies have greatly expanded preclinical and clinical research, and specific therapies have been introduced for three diseases, namely enzyme replacement therapy for Pompe disease (PD), gene expression modulation and gene therapy for Duchenne muscular dys...
Source: Pharmacological Reviews - Category: Drugs & Pharmacology Authors: Tags: Expert Rev Clin Pharmacol Source Type: research
Human Gene Therapy Methods, Ahead of Print.
Source: Human Gene Therapy Methods - Category: Genetics & Stem Cells Authors: Source Type: research
(AFM-T é l é thon) Isabelle Richard's team, a CNRS researcher in an Inserm unit at Genethon, the AFM-Telethon laboratory, has demonstrated the efficacy of gene therapy and determined the effective dose for treating a rare muscle disease, gamma-sarcoglycanopathy, in mouse models of the disease. Based on these encouraging results, published in Molecular Therapy: Methods and Clinical Development, the researchers are preparing a clinical trial.
Source: EurekAlert! - Medicine and Health - Category: International Medicine & Public Health Source Type: news
Several gene therapy trials for Duchenne muscular dystrophy initiated in 2018. Trial decision making is complicated by non-curative, time-limited benefits; the progressive, fatal course; and high unmet needs. ...
Source: Orphanet Journal of Rare Diseases - Category: Internal Medicine Authors: Tags: Research Source Type: research
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