The purpose of SMA Europe is to provide a framework to stimulate collaboration and accelerate translational research pathways in SMA and promote patient care.
Novartis Gene Therapies will initiate SMART, a Phase 3b clinical study to further evaluate the safety and efficacy of Zolgensma® (onasemnogene abeparvovec) in people living with SMA, weighing between 8.5 kg and 21 kg, following a single intravenous (IV) infusion. Clinical data from this study will supplement emerging real-world evidence and use of onasemnogene abeparvovec. […]
In response to our request for regular updates, Roche has shared with us new two-year data from part 2 of its FIREFISH study. These longer-term findings will be presented at the 73rd American Academy of Neurology (AAN) Annual Meeting, which will be held virtually between 17th and 22nd April 2021. FIREFISH is assessing Evrysdi™ (risdiplam) […]
What is apitegromab? Apitegromab (SRK-015) is a muscle-directed therapy that aims to reverse or restrict the muscle atrophy and weakness experienced by people living with SMA. The drug is an antibody that very specifically targets an important protein called myostatin, which restricts muscle mass and strength. Scholar Rock believes that by preventing the activation of […]
As per our request to receive regular updates about the risdiplam clinical development programme, Roche has shared with us new 2-year longer-term data from part 2 of the SUNFISH clinical trial. These data will be presented at the 2021 Muscular Dystrophy Association (MDA) Virtual Clinical & Scientific Conference taking place from 15th to 18th March. […]
We are delighted to share with you that the Committee for Medicinal Products for Human Use (CHMP), of the European Medicines Agency (EMA), adopted a positive opinion recommending the approval of risdiplam for the treatment of 5q SMA, in people 2 months of age and older, with a clinical diagnosis of SMA Type 1, Type […]