News

STAT1d
FDA permits use of Sarepta Therapeutics’ Duchenne therapy in younger patients after short-lived halt
The FDA said it was clearing the way for Sarepta Therapeutics to resume shipments of its gene therapy for Duchenne muscular ...
BioSpace1d
FDA Does 180 on Sarepta, Freeing DMD Gene Therapy for Ambulatory Patients
The swift FDA action removes an overhang from Sarepta and allows Elevidys to return to the market without another safety ...
Fierce Pharma1h
Vinay Prasad departs FDA amid conservative criticism, controversy over Sarepta gene therapy
Vinay Prasad, M.D., has left the FDA, less than three months into the role as director of the Center for Biologics Evaluation ...
STAT5d
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 ...
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 ...
MedPage Today on MSN1d
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 ...
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 ...
Two boys died after a gene therapy. This family won’t give up hope.
The Revell family hoped a gene therapy could buy time for their sons, who have a rare and fatal disease. After two patients ...
Stocktwits on MSN10h
FDA Official Who Halted Sarepta’s Gene Therapy Resigns Amid Backlash Over Access To Duchenne Treatment
Vinay Prasad has stepped down as head of the FDA’s biologics division just two months into the job, following growing ...
Just The News12h
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 ...
Medscape4d
EMA Says No to Duchenne Gene Therapy Elevidys
The European drug authority recommends refusal of marketing authorization after study shows no benefit over placebo.