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SMA News Today is providing live coverage of the MDA Clinical & Scientific Conference taking place March 16-19 in Dallas. Stay tuned to this page for all the latest news.

March 28, 2025 News by Marisa Wexler, MS

MDA 2025: Spinraza treatment stabilizes adult motor function

Treatment with Spinraza (nusinersen) can help stabilize motor function in adults with spinal muscular atrophy (SMA), according to a new analysis. “This study aligns with previously reported findings and suggests that long-term…

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March 27, 2025 News by Marisa Wexler, MS

MDA 2025: SMA gene therapy ‘transformational,’ per Novartis exec

It’s been almost six years since the one-time gene therapy Zolgensma (onasemnogene abeparvovec-xioi) was first approved in the U.S. to treat young children with spinal muscular atrophy (SMA). Now, more than 95%…

March 26, 2025 News by Marisa Wexler, MS

MDA 2025: Apitegromab could fill unmet SMA needs, executive says

The muscle-strengthening therapy apitegromab is being considered for approval by the U.S. Food and Drug Administration (FDA) as an add-on treatment for spinal muscular atrophy (SMA), with a decision expected around the…

March 25, 2025 News by Marisa Wexler, MS

MDA 2025: Potassium channel could be SMA treatment target

Activating a protein called potassium channel Kv2.1 may help improve motor function in spinal muscular atrophy (SMA), according to data from experiments done in mice that point to the protein’s potential as a…

March 24, 2025 News by Marisa Wexler, MS

MDA 2025: High-dose Spinraza safe, effective in DEVOTE study

A higher dose of Spinraza (nusinersen) may be more effective than the currently approved dosing schedule in people with spinal muscular atrophy (SMA) for maintaining motor function, according to data from the…

March 21, 2025 News by Marisa Wexler, MS

MDA 2025: Apitegromab safely improves SMA motor function

Apitegromab, a muscle-strengthening therapy up for approval in the U.S., led to gains in motor function in a clinical trial for people with spinal muscular atrophy (SMA) who took disease-modifying therapies. Data…

March 20, 2025 News by Marisa Wexler, MS

MDA 2025: Evrysdi consistent after 5-year SUNFISH follow-up

After five years of treatment with Evrysdi (risdiplam), people with spinal muscular atrophy (SMA) types 2 and 3 continue to see stabilization and improvement in measures of motor function. That’s according to…

March 19, 2025 News by Marisa Wexler, MS

MDA 2025: SMA kids maintaining Zolgensma benefits 10 years later

Children with spinal muscular atrophy (SMA) given the gene therapy Zolgensma (onasemnogene abeparvovec-xioi) in infancy are maintaining motor milestones after up to a decade of follow-up, according to new long-term clinical trial…

March 19, 2025 News by Marisa Wexler, MS

MDA 2025: SMA gene therapy OAV101 shows acceptable safety

OAV101 IT, a version of the gene therapy Zolgensma (onasemnogene abeparvovec-xioi) that’s administered into the spinal canal, can be safely given to people with spinal muscular atrophy (SMA) who were previously treated…

March 17, 2025 News by Marisa Wexler, MS

MDA 2025: Conference begins, marking another year of progress

The Muscular Dystrophy Association‘s (MDA) annual Clinical & Scientific Conference kicked off over the weekend, bringing together patients, caregivers, researchers, clinicians, academics, advocates, and industry leaders to discuss the latest in science…

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Recent Posts

  • Treatment with AJ201 shows positive signs in SBMA clinical trial
  • Medicines into amniotic fluid may be way to treat SMA before birth
  • Why attitude is important when it comes to accessibility
  • SMA gene therapy GC101 found safe, effective in animals
  • Blood creatinine-to-cystatin-c ratio may track SMA treatment response


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