This page has been translated automatically into English by Google Translate. Always consult with your healthcare team for any medical concerns. All PDFs and printed documents are only available in English.
Treatments & Research
Treatments & Research
SMA Drug Pipeline
The development of all possible SMA treatments being tested in human clinical trials.
Access to Treatment
Information on accessing treatment in the UK and information for those living overseas.
Drugs Currently Being Tested in Clinical Trials
SMA treatments in the pipeline and drugs currently being tested in clinical trials.
Nusinersen (Spinraza)
Access to Biogen’s drug for those who meet the eligibility criteria.
Risdiplam (Evrysdi)
Access to Roche’s drug for those who meet the eligibility criteria.
Zolgensma (Onasemnogene Abeparvovec)
Access to Novartis’ drug for children who have SMA Type 1 and meet the eligibility criteria.
Measures Used to Assess a Drug’s Effectiveness
The tools and measures that may be used as part of collecting information about a drug’s clinical effectiveness.
Clinical, Innovative and Basic Research
New and ongoing projects that have been taking place.
Treatment Approaches
Different approaches that have reached human clinical trial stages.
