New York, March 30, 2026 (GLOBE NEWSWIRE) — The Muscular Dystrophy Association (MDA) celebrates today’s announcement that the U.S. Food and Drug Administration (FDA) has approved a High Dose Regimen of SPINRAZA® (nusinersen) for the treatment of spinal muscular atrophy (SMA). For the SMA community, and for MDA which funded the early science behind the therapy, this milestone reflects not only a new medical option, but decades of perseverance, partnership, and progress. The new regimen, developed by Biogen, is based on findings from the DEVOTE study. The High Dose SPINRAZA was designed to deliver a higher concentration of drug through both the loading and maintenance dosing phases, potentially leading to greater clinical benefit while maintaining a well-established safety profile. Read Biogen’s announcement here.
“The approval of a High Dose Regimen of Spinraza represents an important advancement for people living with SMA. We have long known the life-changing impact of the drug, and now, by optimizing dosing, we have the opportunity to deliver even greater and more sustained benefits to patients across the spectrum of disease.” said Dr. Richard Finkel, a leading SMA expert who has been an investigator in many of the pivotal Spinraza trials from St. Jude’s Children’s Research Hospital. “These results underscore the continued innovation in SMA care and reflect the commitment of the scientific and medical community to improving outcomes for every individual affected by this condition.”
This news is personal for Mindy Henderson, Vice President of Disability Outreach and Empowerment at MDA. Henderson, who lives with SMA, was among the first people in Texas to access Spinraza after its initial approval in 2016. “When I was diagnosed, there were no treatments for SMA, only uncertainty,” Henderson said. “Spinraza changed the course of my life and gave me hope I never thought I would see. The new High Dose Regimen of Spinraza approval is proof that progress doesn’t stop with one discovery, it continues to build, thanks to the persistence of researchers, the investment of organizations like Muscular Dystrophy Association, and the voices of our community.”
That persistence began decades earlier with the pioneering work of Adrian Krainer, PhD, at Cold Spring Harbor Laboratory. Supported by MDA research funding, and in collaboration with Ionis Pharmaceuticals, Dr. Krainer’s lab developed the antisense oligonucleotide approach to SMA that made Spinraza possible. “When we began exploring antisense therapy, it was difficult to imagine how far it could go,” said Dr. Krainer. “Muscular Dystrophy Association’s support at a crucial juncture was instrumental in advancing this science, and it is tremendously gratifying to see how these discoveries continue to evolve, with Spinraza bringing even greater impact to people living with SMA.” Read more of Dr. Krainer’s story in MDA’s Quest Media here.
“This milestone marks an exciting step forward for the SMA community. Spinraza has already transformed the lives of countless families, and the approval of a High Dose Regimen opens the door to even greater improvements in strength, function, and quality of life” said Angela Lek, PhD, Chief Research Officer of MDA. “At the Muscular Dystrophy Association, we are committed to advancing therapies that not only extend life but also maximize independence for people living with neuromuscular diseases. Today’s news reflects the power of continued innovation and collaboration to drive meaningful impact for patients.”
As MDA moves forward together for the next 75 years, today’s approval underscores the organization’s enduring role in advancing therapies for neuromuscular diseases. Since its inception, MDA has invested more than $1.2 billion in research, including over $50 million in spinal muscular atrophy (SMA), helping pave the way for more than 25 FDA-approved treatments in the last decade. Beyond research, MDA drives progress through its advocacy, and nationwide MDA Care Center Network at over 150 top medical institutions, as well as the MDA Resource Center and Gene Therapy Support Network, which provide guidance and support for families living with SMA and more than 300 related neuromuscular conditions. For the SMA community, this milestone represents not only scientific progress, but also a testament to resilience, hope, and renewed possibility.
For more information on SMA, and ongoing support for families and medical professionals, contact the MDA Resource Center by phone 1-833-ASK-MDA1 (1-833-275-6321) or email [email protected].
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About Muscular Dystrophy Association
Muscular Dystrophy Association (MDA) has been at the center of progress for people living with muscular dystrophy, ALS, and over 300 other neuromuscular conditions for 75 years. We unite researchers, clinicians, advocates, and families to speed the pace of discovery, improve access to expert care, and ensure inclusion in every aspect of life. Our mission is simple: give the people we serve the tools and opportunities to live longer, more independent lives. To learn more visit mda.org. Follow MDA on social media on Instagram, Facebook, X, Threads, Bluesky, TikTok, LinkedIn, and YouTube.
- FDA Approves High Dose Regimen of SPINRAZA® for Spinal Muscular Atrophy, Reflecting Progress Made Possible Through Decades of MDA Supported Research