Long-term Spinraza found safe, effective across SMA spectrum
Feb 14, 2024 · The data came from SMArtCARE, a registry that collects information from SMA patients in Germany, Switzerland, and Austria.It’s supported by the companies that market the three available SMA therapies: Novartis, which markets gene therapy Zolgensma, Roche, marketer of Evrysdi, and Biogen.. Patients ranged in age from 16 to 71, with a mean of 36 years.
Oral SBMA therapy shows safety and tolerability in Phase 1 trial
Nov 15, 2023 · Results of the Phase 1 study (ACTRN12622000964718) were presented during the 2023 International Kennedy’s Disease Patient Conference held in London on Nov. 4–5.. Nido also presented preliminary findings in an ongoing and observational study in SBMA patients, which appear to support muscle magnetic resonance imaging (MRI) use in evaluating treatment efficacy in clinical trials and as a ...
SMA Type 4: Symptoms, Treatment, and More - SMA News Today
Feb 2, 2023 · One of the rarest forms of this rare disease, SMA type 4 accounts for less than 1% of all cases. It is also called adult-onset or late-onset SMA, because symptoms appear at a much later age than in other SMA types.. Like the other four key forms of SMA, type 4 is caused by mutations in both copies of the SMN1 gene — one inherited from each biological parent.
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Zolgensma found most effective in presymptomatic infants with SMA. Infants with spinal muscular atrophy (SMA) who are presymptomatic and given the gene therapy Zolgensma in the first six weeks of life have better motor, respiratory, and nutritional …
Apitegromab (SRK-015) for spinal muscular atrophy - SMA News …
Jul 18, 2024 · Apitegromab in clinical trials. Apitegromab has been tested in a Phase 1 study in healthy volunteers which showed positive safety results and indicated the therapy was blocking myostatin activity as designed. This led Scholar Rock to launch the Phase 2 TOPAZ study (NCT03921528) and the Phase 3 SAPPHIRE trial (NCT05156320) in people with …
Spinal Muscular Atrophy: Cardiac Disease – HCP SMA News Today
Spinal Muscular Atrophy (SMA) is an inherited neurodegenerative disease, 1 but it is increasingly clear that the pathology of SMA extends beyond ventral horn spinal cord neurons and includes the heart among other organs. 2 In patients with SMA, tissue levels of SMN1 protein are reduced in heart tissues, 3 and numerous mouse models of SMA exhibit cardiac developmental abnormalities which ...
Specialized equipment for spinal muscular atrophy - SMA News …
Feb 6, 2023 · Wheelchairs are mobile chairs, either manual or powered, for people with spinal muscular atrophy (SMA). Wheelchairs can be pushed by a caregiver or rolled by the person with SMA. Power chairs can be controlled with a joystick, a head array or chin control, and offer options such as postural realignment, and pressure relief.
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Nov 6, 2024 · Accurate genetic testing ‘crucial’ for early diagnosis of SMA: Report. The use of advanced genetic testing techniques helped clinicians in China to identify novel mutations in the SMN1 gene in two unrelated children — and thus correctly diagnose them with spinal muscular atrophy (SMA), according…
Types of spinal muscular atrophy - SMA News Today
Feb 2, 2023 · These forms of SMA range from type 0, which occurs before birth, to type 4, in which symptoms do not start until adulthood.The more common types — 1, 2, and 3 — typically manifest in infancy, or in some cases, up through adolescence. All of these main types are caused by mutations in the gene SMN1, which provides instructions for making a …
AveXis, Developer of Zolgensma, Now Known as Novartis Gene …
Sep 14, 2020 · This decision was based partly on the success of Zolgensma, originally developed by AveXis, as a gene therapy for spinal muscular atrophy (SMA) that is approved in the U.S., Japan, Europe, and Brazil as an intravenous infusion. “Novartis sees tremendous potential in the future of gene therapy, and we’ve seen the impact gene therapy can have on so many lives,” Vas Narasimhan, CEO of ...