News

Roche Pauses Contested Gene Therapy’s Shipments Outside US
Roche Holding AG paused some shipments of the controversial gene therapy Elevidys following a similar action by the embattled ...
FDA Suspends Muscular Dystrophy Drug After 3 Patient Deaths
The U.S. Food and Drug Administration ( FDA) has placed an immediate clinical hold on Sarepta Therapeutics' investigational ...
Sarepta Therapeutics crisis is huge blow to Duchenne families, company
Sarepta rebuffed a call from the Food and Drug Administration to halt all shipments of its gene therapy for Duchenne muscular ...
Sarepta Therapeutics laying off dozens in Ohio, won't comply with FDA request to halt therapy
After the FDA request, Cambridge, Massachusetts-based Sarepta said in a statement that it will continue to ship the therapy ...
LEX 189h
Lemonade stand raising awareness and funds for rare genetic disease
A lemonade stand is a fairly common sight for a hot summer day in July. Outside of Kitchen Concepts in Lexington, a white and yellow stand is doing more than just satisfying thirst.
Sarepta pares gains as report suggests an ‘arduous’ path for Duchenne drug
Sarepta Therapeutics (NASDAQ:SRPT) faces an “arduous and treacherous path” to bring its gene therapy Elevidys back to market, ...
STAT1dOpinion
We are mothers of Duchenne patients. Recent setbacks with Sarepta must not stop progress
As mothers of children with this disease, we have wept helplessly in recent months as friends — fellow members of a club we ...
Fierce Biotech1d
Sarepta LGMD trials all hit by FDA hold amid newly surfaced safety concerns over gene therapy
The Sarepta saga continues, with the FDA slapping a clinical hold across all of the company’s investigational limb girdle ...