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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 ...
Researchers identify molecular ‘cookie cutter’ technique for COVID-19 drug discovery pharmatimes
March 24, 2021
Researchers from the University of Birmingham are aiming to isolate and extract the COVID-19 encounter complex for the identification of drug target sites for the disease.
Evrysdi Trial Demonstrates Improvement of Motor Function in SMA Patients americanpharmaceuticalreview
March 22, 2021
PTC Therapeutics announced two-year data from Part 2 of the SUNFISH trial evaluating Evrysdi™ (risdiplam) in children and adults with Type 2 or Type 3 spinal muscular atrophy (SMA) at the 2021 Muscular Dystrophy Association (MDA) Virtual and ...
NHS England agrees deal with Novartis for access to SMA gene therapy Zolgensma pharmatimes
March 08, 2021
NHS England and Novartis Gene Therapies have signed a ‘landmark’ agreement that will make Zolgensma available for patients on the NHS.
CHMP Adopts Positive Opinion for Evrysdi for Spinal Muscular Atrophy americanpharmaceuticalreview
March 03, 2021
PTC Therapeutics announced the European Medicines Agency (EMA) Committee for Medicinal Products for Human Use (CHMP) has recommended the approval of Evrysdi™ (risdiplam) for the treatment of 5q spinal muscular atrophy (SMA) in patients 2 months and ...
Novartis faces delay in expansion of SMA drug Zolgensma indication pharmaceutical-technology
September 27, 2020
Novartis is facing a delay in expansion of indication for its spinal muscular atrophy (SMA) drug Zolgensma (onasemnogene abeparvovec).
PTC Therapeutics Achieves $20M Roche Milestone contractpharma
August 28, 2020
Payment was triggered by the first commercial sale of Evrysdi in the U.S. which was approved by the FDA on August 7 to treat SMA.
EMA accepts marketing application for Spinal Muscular Atrophy treatment europeanpharmaceuticalreview
August 20, 2020
The European Medicines Agency (EMA) has accepted the marketing authorisation application for Evrysdi™ (risdiplam) as a treatment of spinal muscular atrophy (SMA).
PTC Therapeutics Announces FDA Approval of Evrysdi for Spinal Muscular Atrophy americanpharmaceuticalreview
August 13, 2020
PTC Therapeutics announced the U.S. Food and Drug Administration (FDA) has approved Evrysdi™ (risdiplam), the first at-home, orally administered treatment for spinal muscular atrophy (SMA) in adults and children 2 months and older.

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