FDA nod for Roche drug expands its use to babies with spinal muscular atrophy
Roche drug Evrysdi, one of three FDA-approved treatments for spinal muscular atrophy, is now approved for treating those younger than age 2. The regulatory decision […]
Roche drug Evrysdi, one of three FDA-approved treatments for spinal muscular atrophy, is now approved for treating those younger than age 2. The regulatory decision […]
Roche and PTC Therapeutics’ Evrysdi for spinal muscular atrophy (SMA) has been approved by the FDA for younger children with the rare disease, extending its […]
PTC Therapeutic’s Upstaza gene therapy for patients with genetic disorder AADC deficiency has been recommended for approval in the EU, setting up another test of […]
When it comes to ultra-rare diseases, the smaller the patient population, the bigger the challenges. As a global commercial biopharmaceutical company pioneering therapies for ultra-rare […]
A disparity in UK access to a medicine for Duchenne muscular dystrophy (DMD) has been addressed, after Scotland gave a green light to use of […]
1. BioMarin Reports NDA Submission to the US FDA for Vosoritide to Treat Children with Achondroplasia Published: Aug 20, 2020 | Tags: BioMarin, Reports, NDA, […]
Analysts noted that Evrysdi would be priced significantly lower than Biogen’s Spinraza or Novartis’ Zolgensma. While this will provide a market advantage, it would also […]
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