Financial Post15d
FDA and EMA Accept Applications for Higher Dose Regimen of Nusinersen in SMA
2 SPINRAZA has shown sustained efficacy across ages and SMA types with a well-established safety profile based on data in patients treated up to 10 years, 3,4 combined with unsurpassed real-world ...
Biogen Inc.: FDA and EMA Accept Applications for Higher Dose Regimen of Nusinersen in SMA
Applications are based on data from the DEVOTE study, which demonstrate the potential for the investigational higher dose regimen of nusinersen to advance the treatment of SMA CAMBRIDGE, Mass., Jan. 2 ...
Business Wire9d
Scholar Rock Submits Biologics License Application (BLA) to the U.S. FDA for Apitegromab as a Treatment for Patients with Spinal Muscular Atrophy (SMA)
Scholar Rock plans to also study apitegromab in SMA patients under two ... have been or are continuing to be treated with any currently approved SMN therapy (nusinersen, risdiplam and onasemnogene ...
Nasdaq15d
FDA and EMA Accept Applications for Higher Dose Regimen of Nusinersen in SMA
The higher dose regimen of nusinersen comprises a more ... As a foundation of care in SMA, more than 14,000 individuals have been treated with SPINRAZA worldwide.1 The currently approved 12 ...
Healio4d
FDA receives biologics license application for apitegromab in spinal muscular atrophy
A Boston-area biopharmaceutical firm has submitted a biologics license application to the FDA for its investigational, fully ...
Investing on MSN15d
Ionis stock rises on FDA, EMA nod for SMA drug dose increase
Investing.com -- Shares of Ionis Pharmaceuticals (NASDAQ: IONS) climbed 7% today after the company announced that regulatory agencies in the United States and Europe have accepted applications for a ...
Benzinga.com23d
Scholar Rock Sets Sights on $2 Billion Apitegromab Revenue Amid Spinal Muscular Atrophy Market Growth
Detailed results from the phase 3 SAPPHIRE trial, which involves patients with non-ambulatory type 2/3 SMA treated with Spinraza (nusinersen) or Evrysdi (risdiplam), are expected to be presented ...