News

MedPage Today on MSN4d
Shipments of Duchenne Gene Therapy to Resume After FDA Review of Patient Death
Sarepta's therapy has been under scrutiny from regulators after two teenage boys died earlier this year from acute liver ...
Amazon S3 on MSN3d
The story of Sarepta's Duchenne gene therapy
The last couple of weeks has been particularly distressing for Duchenne muscular dystrophy patients and their families. The ...
STAT8d
For many Duchenne families, halt to gene therapy is heartbreak upon heartbreak
As gene therapy maker Sarepta Therapeutics tangles with FDA over its Duchenne treatment, patients and their families are ...
Gene therapy maker Sarepta tells FDA it won’t halt shipments despite patient deaths
Drugmaker Sarepta Therapeutics says it won’t comply with a request from U.S. regulators to halt all shipments of its gene ...
Just The News3d
FDA allows distribution of muscular dystrophy drug again after public criticism
The agency’s now-reversed decision to halt distribution of a gene therapy for children suffering from muscular dystrophy ...
Parents call on FDA for path forward after Duchenne's gene therapy pause | Morning in America
The Food and Drug Administration has paused distribution of the gene therapy Elevidys following the death of a third patient ...
BioPharma Dive8d
Sarepta woes mount as Duchenne gene therapy knocked back in Europe
The European Medicines Agency determined testing failed to prove Elevidys’ benefit and issued a negative opinion that could ...
FDA asks Sarepta to stop shipping Duchenne gene therapy
The company refused the FDA’s request and will continue shipping its therapy, Elevidys, to Duchenne patients who can still ...
FDA opens probe into mysterious death linked to gene therapy
The Food & Drug Administration is investigating the death of an 8‑year‑old following Elevidys gene therapy for Duchenne ...
Medscape7d
EMA Says No to Duchenne Gene Therapy Elevidys
The European drug authority recommends refusal of marketing authorization after study shows no benefit over placebo.
BioPharma Dive17d
Sarepta to lay off about 500 employees after Duchenne gene therapy setbacks
On a conference call, CEO Doug Ingram said the decision to cut 36% of the company's staff and halt several drug programs was ...