Update on PTC’s SMA trials

Published Date
03/10/2018
Author
Sofia Nnorom
Category
Research
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PTC Therapeutics has released preliminary data from its ongoing clinical studies, FIREFISH and SUNFISH. These studies are investigating whether RG7916 (risdiplam) could be a potential treatment for spinal muscular atrophy (SMA).

So far, the FIREFISH study has shown that infants with Type 1 SMA treated with RG7916 have reached new developmental milestones (e.g head control and sitting). The SUNFISH study – which is focussed on people with Type 2/3 SMA – has also shown that RG7916 can improve motor function.

No severe adverse events have been reported to date, suggesting that the drug is safe and well-tolerated.

For further information please read PTC Therapeutics’ press release.

If you have any questions about this news story or any other SMA research, please contact the MDUK Research Line on 020 7803 4813 or email research@musculardystrophyuk.org.

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