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Biogen Exercises Option for Exclusive License to BIIB115 contractpharma
January 04, 2022
BIIB115 is an investigational antisense oligonucleotide (ASO) in development for spinal muscular atrophy.
China increases accessibility to rare disease treatment NMPA/Xinhua
December 24, 2021
Two-and-half-year-old Xuanxuan survived a rare kidney disease in early December, thanks to fast-track approval of the transplant.
Roche awarded 2022 Drug Discovery of the Year for Evrysdi® EuropeanPharmaceuticalReview
December 07, 2021
The British Pharmacological Society has given the team of scientists who discovered and developed Evrysdi® (risdiplam) the Drug Discovery of the Year Award 2022.
Novartis Announces New Phase 3 Study for Spinal Muscular Atrophy Treatment in Elderly Patients americanpharmaceuticalreview
August 04, 2021
Novartis announced that the U.S. FDA determined that OAV-101 intrathecal (IT) clinical trials for spinal muscular atrophy (SMA) patients may proceed, thereby lifting the partial clinical trial hold initiated in October 2019.
Roche’s Evrysdi significantly improves lives of babies with SMA europeanpharmaceuticalreview
August 02, 2021
The Phase II study of Evrysdi showed significant improvements in survival and motor milestones in babies with type 1 spinal muscular atrophy (SMA).
Expanded access for Zolgensma following final NICE guidance pharmatimes
July 12, 2021
The National Institute for Health and Care Excellence (NICE) has published its final guidance on Novartis’ spinal muscular atrophy (SMA) gene therapy Zolgensma.
Zolgensma shows promise in presymptomatic SMA patients pharmatimes
June 22, 2021
Novartis’ gene therapy Zolgensma has shown benefit for presymptomatic spinal muscular atrophy (SMA) patients, according to new data presented at the annual European Academy for Neurology (EAN) virtual congress.
NICE draft guidance does not recommend Evrysdi for SMA pharmatimes
June 04, 2021
The UK National Institute for Health and Care Excellence (NICE) has announced that it does not recommend Roche’s Evrysdi for the treatment of spinal muscular atrophy (SMA) in draft guidance.
NICE expands access for SMA drug Spinraza pharmatimes
May 07, 2021
The UK’s National Institute for Health and Care Excellence (NICE) has extended the clinical eligibility criteria for Biogen’s spinal muscular atrophy (SMA) drug Spinraza following a review of data collected as part of a managed access agreement (MAA).
Evrysdi from ROCHE gets EC nod as home treatment for spinal muscular atrophy expresspharma
March 31, 2021
Roche announced that the European Commission (EC) has approved Evrysdi (risdiplam) for the treatment of 5q spinal muscular atrophy (SMA) in patients two months of age and older, with a clinical diagnosis of SMA Type 1, Type 2 or Type 3 or with one to ...

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