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Drugs Currently being tested in Clinical Trials

Find out what clinical trials are now recruiting in the UK by clicking here.

To register your interest in future trials, contact the Patient Registry.     

For an overview of progress, please see the Drug Pipeline.

Some of the drugs currently being tested in clinical trials:

AVXS-101 (AveXis)

AVXS-101 uses harmless, genetically-engineered viruses to increase SMN protein levels. It has recently received “Breakthrough Therapy” status. AveXis, the pharmaceutical company developing AVXS-101, has released results from the completed Phase I trial of the gene therapy drug.

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CK-2127107 (Cytokinetics)

CK-2127107 is a drug able to increase the force generated by skeletal muscle and delay the onset and extent of muscle fatigue. Cytokinetics, the pharmaceutical company developing CK-2127107, recently announced that it is starting to enrol individuals with SMA Types 2, 3 or 4 in their phase 2 clinical trial in North America, where the drug was granted Orphan Drug status in May 2017.

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Olesoxime (Trophos / Roche) 

Olesoxime is a neuro-protective drug that supports the nervous system under stressful conditions. In January 2015, a Phase II clinical trial with young people aged 3 – 25 with SMA Type 2 or 3, indicated that Olesoxime is safe, well tolerated, and able to positively impact disease progression. At the same time, Roche pharmaceutical company agreed to purchase the small, late-stage clinical drug testing company, Trophos that originally developed the drug.

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RG7916 (Roche) 

RG7916, developed by Roche, is a small molecule that targets and encourages the SMN2 ‘back-up’ gene to produce a greater amount of functional SMN protein, which is lacking in people with SMA. In January 2017, the US Food and Drug Administration (FDA) granted RG7916 Orphan Drug Status.

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