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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.

Image shows female scientist in a laboratory using a microscope.

Drugs Currently Being Tested in Clinical Trials

SMA treatments in the pipeline and drugs currently being tested in clinical trials.

Image shows the Nusinersen (Spinraza) treatment box and bottle.

Nusinersen (Spinraza)

Access to Biogen’s drug for those who meet the eligibility criteria.

Image shows the Risdiplam (Evrysdi) bottle and box.

Risdiplam (Evrysdi)

Access to Roche’s drug for those who meet the eligibility criteria.

Image shows the Zolgensma bottle and box.

Zolgensma (Onasemnogene Abeparvovec)

Access to Novartis’ drug for children who have SMA Type 1 and meet the eligibility criteria.

Measures of drug effectiveness (website card)

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.

Image shows a close-up of a microscope.

Clinical, Innovative and Basic Research

New and ongoing projects that have been taking place.

Image shows neurons firing.

Treatment Approaches

Different approaches that have reached human clinical trial stages.