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Zolgensma — given alone, after, or in combination with another spinal muscular atrophy (SMA) disease-modifying therapy — leads to clinically meaningful motor improvements in children older than six months, according to early real-world data from the RESTORE registry. Reported side effects were also consistent with Zolgensma’s previously described…
Editor’s note: The SMA News Today team is providing in-depth coverage of the 2021 MDA Virtual Clinical and Scientific Conference, March 15–18. Go here to read the latest stories from the conference. Six months of treatment with apitegromab, Scholar Rock’s muscle-directed therapy…
Editor’s note: The SMA News Today team is providing in-depth coverage of the 2021 MDA Virtual Clinical and Scientific Conference, March 15–18. Go here to read the latest stories from the conference. The Phase 2 RAINBOWFISH trial of Evrysdi (risdiplam) in newborns up to 6 weeks old with a…
Editor’s note: The SMA News Today team is providing in-depth coverage of the 2021 MDA Virtual Clinical and Scientific Conference, March 15–18. Go here to read the latest stories from the conference. Treatment with Evrysdi (risdiplam) is safe and increases the levels of SMN — the missing protein in spinal muscular…
Michael and I have been working together for the past couple of years, and while speaking with each other about my March HCP article, we thought that doing a question-and-answer article would be interesting. Michael would ask me 5 questions, and I would ask him 5 questions. We hope that…
PTC Therapeutics and the Spinal Muscle Atrophy (SMA) Association will collaborate in funding research into regenerative medicine, and into SMA in particular. With the advent of disease-modifying SMA therapies such as Evrysdi (risdiplam) and Spinraza (nusinersen), regenerative medicine — which aims to restore damage caused by…
Eight weeks of treatment with investigational add-on oral therapy reldesemtiv safely improved motor and respiratory strength in adolescents and adults with spinal muscular atrophy (SMA), according to final data from a Phase 2, hypothesis-generating clinical trial. These findings support further clinical testing of reldesemtiv, designed to aid skeletal muscle…
The gene therapy Zolgensma was added to the list of medications available to eligible spinal muscular atrophy (SMA) type 1 patients through England’s and Scotland’s national health services (NHS). With a reported list price of £1.79 million ($2.48 million) per dose — the most expensive yet — Novartis’ Zolgensma uses…
Many diseases have their own awareness color — breast cancer is pink, muscular dystrophy is green, and AIDS is red, for example — but what’s the significance of pink, green, blue, and purple lights side-by-side? These are the colors most often used to represent Rare Disease Day. This…
Adults with spinal muscular atrophy (SMA) usually do not experience weakness or impairment of any sort in the muscles controlling their eye movements, a recent study reported. When such impairments are present, they should prompt physicians to re-evaluate their initial diagnosis and consider the possibility that a patient may…
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