Deaths associated with Astellas Pharma’s gene therapy program for patients with X-linked Myotubular Myopathy underscore some of the risks of developing potential one-and-done treatment options for serious diseases.
The U.S. Food and Drug Administration approved Vyvgart (efgartigimod alfa-fcab) for the treatment of generalized myasthenia gravis (gMG) in adult patients positive for anti-acetylcholine receptor (AChR) antibody.
A patient treated with Astellas Pharma’s experimental gene therapy for patients with X-linked Myotubular Myopathy (XLMTM) died after reports of a serious adverse event following treatment with AT132.
Astellas Pharma paused the screening and dosing of an investigational gene therapy for patients with X-linked Myotubular My-opathy (XLMTM) following a serious adverse event report in a trial participant.
The U.S. Food and Drug Administrationlifted a clinical hold on Audentes Therapeutics’ ASPIRO trial studying AT132 as a potential treatment for X-linked myotubular myopathy (XLMTM), a rare neuromuscular disease.
Novartis said new interim data from an ongoing phase clinical trial for the company’s $2.1 million-per-patient gene therapy Zolgensma showed spinal muscular atrophy patients experienced significant therapeutic benefit.
A third pediatric patient involved in a gene therapy trial for a rare, neuromuscular disease conducted by Audentes Therapeutics died.
COVID-19 tied to muscle-weakening disease; vaping risk documented
Annals of Internal Medicine, Brain, Coronavirus Disease 2019 (COVID-19), Coronavirus Infections, Coronavirus Vaccines, Doctors, E-Cigarettes, Illnesses, Immune Response, Journal of Adolescent Health, Masks, Medical Equipment, Muscle Wasting Disorders, Myasthenia Gravis (MG), Nervous System, Neuromuscular diseases, Researchers, Science, Scientific studies, VapingA roundup of some of the latest scientific studies on the novel coronavirus and efforts to find treatments and vaccines for Covid-19, the illness caused by the virus.
San Francisco-based Audentes Therapeutics reported two deaths in the company’s gene therapy clinical trial for AT132 for X-linked myotubular myopathy.
Roche company Genentech presented one-year data from FIREFISH Part 2, a pivotal trial of risdiplam in infants one to seven months old with symptomatic Type 1 spinal muscular atrophy (SMA).