Keyword: Sarepta

A trial of Sarepta’s gene therapy for muscular dystrophy was placed on clinical hold by the FDA but could be upgraded to a pivotal study as a result.

The initiative taps into the networks Invitae has built up to connect sponsors to patients and the clinicians responsible for their care.

Sarepta licenses five new gene therapies to its muscular dystrophy portfolio from Myonexus—and takes an option on buying the company.

Buoyed by safety data and evidence of exon-skipping activity, Daiichi is pushing ahead with further development of the antisense oligonucleotide.

Pfizer has dosed the first patient in a trial of a muscular dystrophy gene therapy, acquired as part of its $700 million takeover of Bamboo in 2016.

The latest analysis links the utrophin modulator to a statistically significant decline in muscle inflammation in boys with DMD.

Sarepta is hoping to avoid the data-quality controversy that arose over its Duchenne muscular dystrophy drug Exondys51 last year with its next-generation drug golodirsen.

A panel of experts convened by the FDA has overwhelmingly knocked back PTC Therapeutics’ filing for approval of ataluren.

Wednesday's shot of your news of note.

Sarepta offers Exondys 51 outside of the U.S. through a managed access program run by Clinigen, as an EMA decision is expected next year.