Home » Sma type 1, improve long-term motor functions with risdiplam

Sma type 1, improve long-term motor functions with risdiplam

by admin
Sma type 1, improve long-term motor functions with risdiplam

More than 5,000 patients in the world have now received risdiplam oral therapy against spinal muscular atrophy (SMA), recently approved in Italy. And the arrival of new data from ongoing clinical trials confirms its effectiveness. The latest, in chronological order, are those of the FIREFISH study just presented at the 14th congress of the European Pediatric Neurology Society (EPNS). They affect children with type 1 AMS, the most severe form of the degenerative disease affecting motor neurons. With risdiplam these children are able to maintain or improve motor functions and 91% of treated patients were alive three years after treatment (58). In parallel, during the study period, adverse events decreased, confirming the efficacy and safety of the drug.

Sma, the first oral therapy at home approved

by Mara Magistroni


In SMA type 1 without treatment, these children are never able to sit without support. However, the arrival of risdiplam, which works by increasing the production of the SMN protein, essential for the survival and health of motor neurons, has changed the history of the disease. Not only in older patients with forms of SMA 2 and 3, but also in smaller ones, affected by SMA type 1, the most severe form, which affects already in the first months of life, compromising movement skills, swallowing and breathing.

Children included in the FIREFISH study were 1 to 7 months old at enrollment. Data presented at the EPNS on long-term use of the drug – administered daily at home in liquid form – show that children treated with risdiplam maintained or continued to experience improvements in the ability to sit without support between 24 and 36 months. for 5 and 30 seconds. None of those who had acquired this ability lost it in the three years of treatment. Improved and generally maintained other motor functions, such as the ability to keep the head straight, rotate while seated, walk with support, or stand with support. Most of the children treated also maintained the ability to feed orally and swallow until the 36th month.

See also  Waiting lists, delays mainly affect the elderly

Sma, oral therapy is effective for a long time

by Dario Rubino



During the course of treatment, the adverse effects, more or less serious, also decreased (we are talking about effects attributable to the disease and not to the treatment), as well as hospitalizations. To the point that, commenting on the results, Levi Garraway, Chief Medical Officer and Head of Global Product Development at Roche, said: “These long-term results in children treated with risdiplam are extremely encouraging: the vast majority have improved or maintained functions. motor after three years. Normally, without treatment, these children would not survive beyond two years of age. The data supporting the efficacy of risdiplam continues to grow across a broad spectrum of individuals, including children suffering from one of the most severe forms of SMA ”.

You may also like

Leave a Comment

This site uses Akismet to reduce spam. Learn how your comment data is processed.

This website uses cookies to improve your experience. We'll assume you're ok with this, but you can opt-out if you wish. Accept Read More

Privacy & Cookies Policy