sma
Rare diseases – spinal muscular atrophy
January 30, 2026Medical Communications gene therapy, medical communications, muscle and motor function, muscle weakness, rare diseases, sma
Spinal muscular atrophy (SMA) is a rare disease characterised by voluntary muscle weakness that can lead to difficulties in activities …
Novartis shares new data for Zolgensma in children with SMA
March 5, 2024Musculo-skeletal disorder, Novartis, Zolgensma, sma, spinal muscular atrophy
Novartis has announced new data to continue the support of the clinical benefits of Zolgensma (onasmenogene abeparvovec), for the treatment …
Study shows significant improvement of survival in infants with SMA
July 29, 2021sma
Results from the Evrysdi part 2 global FIREFISH study show significant improvement in survival and motor milestones in babies with …
NICE finds more SMA patients eligible for £75,000 a vial drug
May 4, 2021Manufacturing and Production, Sales and Marketing NICE, sma
NICE has announced that more people with the rare genetic disorder spinal muscular atrophy (SMA) are to benefit from nusinersen, …
Roche’s SMA treatment Evrysdi approved by European Commission
March 30, 2021Manufacturing and Production EMA, European Commission, PTC Therapeutics, Roche, sma, spinal muscular atrophy
The European Commission has granted marketing authorisation to Roche and PTC Therapeutics’ Evrysdi, for the treatment of patients aged 2 …
Roche and PTC’s SMA treatment shows positive trial results
March 16, 2021Manufacturing and Production PTC, PTC Therapeutics, Roche, sma, spinal muscular atrophy
Roche and PTC Therapeutics’s Evrysdi treatment for children and adults with Type 2 or Type 3 spinal muscular atrophy (SMA) …
Novartis plans giveaways of world’s most expensive drug
December 19, 2019Business Services Novartis, Zolgensma, sma
Novartis plans to giveaway 50 doses of the world’s costliest single-dose treatment, Zolgensma, for babies under 2 years old, with …
Novartis exec pushed out over Zolgensma data scandal denies wrongdoing
August 21, 2019Research and Development AveXis, FDA, Novartis, Zolgensma, data scandal, pharma, sma
Dr Brian Kaspar, the Novartis exec pushed out of AveXis over the Zolgensma data scandal, has said in a statement …
Novartis’ $2.15m SMA drug Zolgensma becomes most expensive FDA-approved therapy
May 28, 2019Manufacturing and Production AveXis, FDA, Zolgensma, access, drugs, pricing, sma
The FDA has approved Novartis subsidiary AveXis’ $2.15 million gene therapy Zolgensma for children with spinal muscular atrophy (SMA). The …
NHS England agree to pay for Biogen’s $750,000 a year SMA treatment Spinraza
May 15, 2019Research and Development Biogen, NHS England, Spinraza, UK, pharma, sma, spinal muscular atrophy
NHS England has agreed to pay an undisclosed price for Biogen’s spinal muscle atrophy (SMA) treatment Spinraza. England’s National Health …
Protesters in Manchester call on NICE to approve Biogen’s $750,000 drug Spinraza
September 11, 2018Business Services, Manufacturing and Production, Medical Communications, Research and Development, Sales and Marketing NICE, Spinraza, approval, protest, sma
Protesters in Manchester have called on the National Institute of Health and Care Excellence (NICE), to overturn their rejection of …
Roche buys French biotech for €470 million
January 19, 2015Sales and Marketing Foundation, Genentech, Orphan, Roche, TRO19622, olesoxime, sma, trophos
Roche is buying France-based biotech Trophos in a deal worth up to €470 million in order to gain access to …
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