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.
A 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).