Top 10 SMA news stories of 2025
Hot topics ranged from Zolgensma’s lasting benefits to clinical trial data
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Throughout 2025, the team at SMA News Today has brought our audience the latest advances in spinal muscular atrophy (SMA), from emerging treatments and clinical trial updates to real-world research.
As the year comes to a close, we’ve taken a look back at the top 10 stories that captured our readers’ attention. We look forward to continuing to support and inform the SMA community in 2026.
No. 10 – Apitegromab moves closer to FDA approval as add-on therapy
Scholar Rock was planning to have apitegromab, its muscle-directed add-on treatment for SMA, commercially available in the U.S. before the end of this year. The U.S. Food and Drug Administration (FDA) was expected to decide on whether to approve apitegromab in September. The therapy is designed to improve muscle strength and function in people with SMA by blocking myostatin, a natural inhibitor of muscle growth. However, the FDA ultimately rejected the application, citing manufacturing issues. Scholar Rock is therefore planning to seek the therapy’s approval again in 2026.
No. 9 – Biogen pushes forward with salanersen, next-gen therapy
Biogen is stepping up efforts to develop salanersen, an antisense oligonucleotide therapy designed to boost SMN protein production in people with SMA. Early results from a Phase 1 clinical trial have been described as “encouraging,” prompting the company to work closely with regulators on shaping the program’s next steps. In a written interview, Stephanie Fradette, Biogen’s head of the neuromuscular development unit, highlighted how salanersen leverages the same mechanism of action as Spinraza (nusinersen), while aiming for greater durability and potency with once-yearly dosing.
No. 8 – Evrysdi leads to steady functional gains in SMA types 2 and 3
Genentech’s Evrysdi (risdiplam) continued to show sustained functional benefits in people with SMA types 2 and 3. Data from SUNFISH, a Phase 2/3 trial, show that after five years of treatment, motor function remained stable or improved, reinforcing Evrysdi’s long-term effectiveness and safety. Improvements translate into daily life, and better motor function scores in the arms and hands can reflect greater ability to “lift an object” or “bring something to your mouth,” said Laurent Servais, MD, PhD, who was a presenter at the Muscular Dystrophy Association‘s (MDA) annual Clinical & Scientific Conference.
No. 7 – Gene therapy GC101 shows promise for children with SMA types 2, 3
A small clinical study in China found that the investigational gene therapy GC101 was generally safe in children and adolescents with SMA types 2 and 3. Results from nine participants showed improved or stabilized motor function, with a clinically significant response in all three children treated when they were younger than 2. Notably, five patients who had been showing motor regression regained gross motor skills after treatment, such as jumping or walking with assistance. GC101 is being explored in Phase 1/2 trials in SMA types 1, 2, and 3.
No. 6 – Expert shares when to consider surgery for SMA-related scoliosis
For people with SMA facing scoliosis, or a sideways curvature of the spine, surgery is often a major consideration. In an interview, Arun Hariharan, MD, a pediatric orthopedic and spine surgeon specializing in complex spinal deformities and care for children with neuromuscular conditions, discussed topics such as when surgery should be conducted, what symptoms may ease with the procedure, how an SMA patient can plan for the surgery, and the major concerns that families may have.
No. 5 – Novartis exec says Zolgensma has transformed SMA care
The one-time gene therapy Zolgensma (onasemnogene abeparvovec-xioi) has been “transformational” in SMA care, as more than 95% of eligible babies have received the treatment. That’s according to Daniel Grant, PhD, vice president and global program head at Novartis, who spoke with SMA News Today at the annual MDA meeting. Grant discussed the “very high expectations” resulting from treating SMA very early in life, but he also mentioned Phase 3 data for Itvisma (onasemnogene abeparvovec-brve), a one-time gene therapy that was approved by the FDA late this year for SMA patients 2 years and older.
No. 4 – High-carb diet may help restore healthier metabolism in SMA
A new study suggests a high-carbohydrate diet may help normalize metabolic changes in people with SMA, as these patients experience metabolic alterations due to changes in how their bodies utilize fatty acids for energy. SMA patients had high levels of free fatty acids, which can be burned for energy, and of ketone bodies, byproducts of breaking down fats for energy. This suggests an abnormal upregulation of all available metabolic pathways to produce energy, according to scientists.
No. 3 – Amino-acid imbalance identified in SMA
People with SMA have notable disruptions in the levels of certain amino acids (the building blocks of proteins) that influence neurological function, a study reveals. These imbalances were seen in both SMA patient samples and in a mouse model. Some of the alterations in patients were partially corrected by treatment with Spinraza. In mice, treatment with D-serine, an amino acid, improved motor function. Collectively, the results warrant more studies into potential strategies for combination therapies.
No. 2 – High-dose Spinraza deemed safe and effective in DEVOTE trial
Results presented at the MDA annual meeting showed that a higher dose regimen of nusinersen (the active ingredient in Spinraza) was safe and potentially more effective than the approved dosing schedule in maintaining motor function, among other benefits, in people with SMA. The DEVOTE clinical trial tested a new dosing schedule where patients received two 50 mg loading doses 14 days apart, followed by 28 mg maintenance doses every four months. However, the FDA rejected an application to approve this regimen later in the year, requesting an update to technical information. Biogen stated at the time that it planned to resubmit the application.
No. 1 – Zolgensma shows lasting benefits a decade after treatment
A decade-long follow-up of Zolgensma treatment indicates that many children with SMA continue to maintain motor function milestones. At the MDA meeting, trial data showed that all children given the therapeutic dose of Zolgensma were alive without the need for permanent ventilation. All were able to sit without support, five could stand with assistance, and two were able to walk unassisted.
We hope that our reporting in 2025 has offered valuable insights to the SMA community. We look forward to continuing to share important news and developments in 2026. Warmest wishes for a happy new year to all our readers.
