Sarepta | pharmaphorum

Saturday 23 November 2024

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Sarepta

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Sarepta DMD gene therapy could improve patients' motor function

Sarepta DMD gene therapy could improve patients' motor funct...

Sarepta Therapeutics announced potentially game-changing results from its Duchenne muscular dystrophy gene therapy candidate, with a small phase 1 trial showing an improvement in patients’

News

Sarepta dives deeper into gene therapy

Sarepta dives deeper into gene therapy

Sarepta Therapeutics has handed $30 million to Lacerta in exchange for a group of gene therapies, including a Pompe disease candidate.

News

DNA sequence

Summit shares plunge nearly 80% after DMD drug fails

Summit Therapeutics has axed development of its Duchenne muscular dystrophy drug ezutromid after a spectacular failure in phase 2, causing the UK biotech's shares to plunge.

News

NICE1

Pharma collaborates to improve chances of NICE backing Duche...

Project will initially focus on DMD but could benefit other rare diseases

News

Contrasting fortunes for Duchenne rivals, as PTC fails and S...

Two rivals in the emerging Duchenne muscular dystrophy (DMD) therapy area have experienced contrasting fortunes this week.

News

FDA-sign

PTC Therapeutics fails to convince FDA panel about DMD drug

FDA advisers have voted against approval for PTC Therapeutics’ Duchenne drug Translarna because of doubts about its efficacy.