spinal muscular atrophy
Five Facts about spinal muscular atrophy
January 28, 2026Medical Communications Five Facts, Neurology, early diagnosis, five types, gene therapy, muscle weakness, spinal muscular atrophy
1 Spinal muscular atrophy (SMA) is a genetic disease involving the loss of motor neurons in the spinal cord.1 Its …
Spike in searches relating to spinal muscular atrophy following Jesy Nelson disclosure
January 19, 2026Medical Education Neurology, spinal muscular atrophy
New research from the Centre for Surgery has shown a significant rise in Google searches relating to spinal muscular atrophy …
Biogen’s high-dose Spinraza regimen receives European Commission approval for spinal muscular atrophy
January 19, 2026Research and Development Biogen, Neurology, Spinraza, spinal muscular atrophy
Biogen’s Spinraza (nusinersen) has received approval from the European Commission (EC) as a new high-dose regimen for the treatment of …
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 …
NMD Pharma begins phase 2 trial for spinal muscular atrophy treatment
September 27, 2023Research and Development Musculo-skeletal disorder, NMD Pharma, clinical trial, spinal muscular atrophy
NMD Pharma has announced that the first patient has been dosed in its phase 2 clinical trial for its CIC-1 …
EC approves Roche’s Evrysdi for babies under two months with SMA
August 30, 2023Research and Development European Commission, Evrysdi, Musculo-skeletal disorder, Roche, spinal muscular atrophy
Roche has announced that the European Commission (EC) has approved the extension of Evrysdi’s marketing authorisation in the EU to …
Roche’s Evrysdi drug for SMA shown to significantly increase survival
April 15, 2021PTC Therapeutics, Roche, pharma, pharma news, spinal muscular atrophy
Roche’s Evrysdi therapy for the treatment of children with type 1 spinal muscular atrophy (SMA) has been shown to increase …
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) …
NICE recommends £1.79m gene therapy for spinal muscular atrophy in babies
March 8, 2021Sales and Marketing NICE, spinal muscular atrophy
NICE is set to approve a new and potentially curative one-off gene therapy for babies with the rare genetic disorder …
Novartis unveils strong Phase 3 efficacy for one-time therapy Zolgensma in paediatric spinal muscular atrophy
October 1, 2020Research and Development Novartis, Zolgensma, spinal muscular atrophy
Novartis has lifted the curtain on new Phase 3 data from an ongoing trial of 33 participants on the efficacy …
Genentech’s Evrysdi becomes first FDA-approved oral treatment for spinal muscular atrophy
August 10, 2020Sales and Marketing Evrysdi, FDA, Genentech, Roche, spinal muscular atrophy
Genentech’s Evrysdi (risdiplam) has secured FDA approval for the treatment of spinal muscular atrophy (SMA), making it the first oral therapy …
Novartis reveals hard-hitting five-year data for its gene therapy Zolgensma in spinal muscular atrophy
March 25, 2020Research and Development Novartis, Zolgensma, gene therapy, pharma, spinal muscular atrophy
Novartis has revealed five-year data on its gene therapy Zolgensma, boasting that the one-time infusion demonstrated “rapid, significant and clinically …
Japan approves Novartis’ Zolgensma for spinal muscular atrophy in patients under two years old
March 19, 2020Sales and Marketing Japan, Novartis, Zolgensma, pharma, spinal muscular atrophy
Novartis has revealed that its gene therapy Zolgensma (onasemnogene abeparvovec) has secured approval from Japan’s Ministry of Health, Labour and …
Roche’s risdiplam hits main goal in infants with spinal muscular atrophy
January 23, 2020Research and Development Roche, pharma, risdiplam, spinal muscular atrophy
Roche has announced the success of its survival motor neuron-2 (SMN2) splicing modifier risdiplam in a new trial investigating its …
