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Treatments & Research
Treatments & Research
Research & Treatment Overview
Including our Drug Pipeline showing the progress of SMA treatments tested in human clinical trials.
Access to Treatment
Information on accessing treatment in the UK and information for those living overseas.
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 (IV – Onasemnogene Abeparvovec)
Access to Novartis’ drug for children who have SMA Type 1 and meet the eligibility criteria.
Intrathecal Onasemnogene Abeparvovec (OAV101 IT)
Now approved for use in the USA, this treatment is now on the path to be considered for use in England
Drugs Currently Being Tested in Clinical Trials
SMA treatments in the pipeline and drugs currently being tested in clinical trials.
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.
Open Research Studies and Reports
New and ongoing projects that have been taking place.
