Sarepta says early filing for DMD gene therapy is back on
Sarepta is pressing forward with a bold plan to file with the FDA for accelerated approval of its gene therapy SRP-9001 for Duchenne muscular dystrophy […]
Sarepta is pressing forward with a bold plan to file with the FDA for accelerated approval of its gene therapy SRP-9001 for Duchenne muscular dystrophy […]
New data on Sarepta’s gene therapy for Duchenne muscular dystrophy (DMD) has gone a long way towards building confidence in its efficacy – but have […]
Shares in Sarepta came under pressure after the biotech said it had temporarily halted a clinical trial of its new-generation Duchenne muscular dystrophy candidate, after […]
Just days after dosing its first patient in a Duchenne muscular dystrophy trial, US biotech PepGen has pushed the button on an initial public offering […]
The development of gene therapies for Duchenne muscular dystrophy has proved to be challenging, but one of the key players – Sarepta – thinks it […]
Pfizer has called a halt to a clinical trial of its gene therapy for Duchenne muscular dystrophy as it investigates the unexpected death of a […]
Shots: The SRP-9001 program consists of 4 studies i.e., SRP-9001-301/103/102/101 that evaluates SRP-9001 in patients with DMD In studies 101 & 102, SRP-9001-treated patients showed […]
The unexpected approval and lofty pricing of Biogen’s Alzheimer’s therapy Aduhelm is clearly the biopharma event of the year so far, but what are the […]
BeiGene Reports Results of Tislelizumab + CT in P-III RATIONALE 309 Study as 1L Treatment for Recurrent or Metastatic Nasopharyngeal Cancer Published: May 21, 2021 […]
Shots: Sarepta reported positive 12wks. expression & safety results from the first 11 participants enrolled in Study SRP-9001-103. The study uses commercially representative SRP-9001 Patients […]
The first UK patient has enrolled on Pfizer’s phase 3 trial for its Duchenne muscular dystrophy (DMD) gene therapy. Pfizer’s CIFFREO study began in the […]
With clinical holds lifting there has been more investment and clinical progress in the gene therapy for muscular disorders space than ever before, yet the […]
Almirall and Tyris Collaborate to Develop Next Generation Gene Therapies Published: Jan 7, 2020 | Tags: Almirall, Tyris, Collaborate, Develop, Next Generation, Gene Therapies Sarepta […]
Shots: The Study 102 involves assessing of SRP-9001 vs PBO in 41 patients in a ratio (1:1) with DMD aged 4-7yrs. Results: @12wks. post treatment, […]
Sarepta chief commercial officer Bo Cumbo has left to head up gene therapy venture – AavantiBio – with $107 million in backing from his former […]
Sobi & Selecta Report Results of SEL-212 in P-II COMPARE Study for Chronic Refractory Gout Published: Oct 1, 2020 | Tags: Chronic Refractory Gout, COMPARE […]
The FDA has approved a new therapy for the rare muscle wasting disease Duchenne muscular dystrophy (DMD) as Japan’s NS Pharma takes on Sarepta and […]
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