Data presented at the 2026 Muscular Dystrophy Association meeting could have readthroughs to companies developing therapies for spinal muscular atrophy, Duchenne muscular dystrophy and Becker muscular dystrophy.
The 2026 Muscular Dystrophy Association conference landed in Orlando, Florida this year, bringing with it some of the newest developments and cutting-edge research in the field.
As the congress wrapped up on Wednesday, BioSpace looks at some of the most compelling presentations that are likely to make a big impact on biopharma—and patients.
Biogen’s Oligonucleotide SMA Therapy Builds Momentum For Phase 3 Program
Biogen’s antisense oligonucleotide salanersen significantly lowered levels of a key disease biomarker in a Phase 1b spinal muscular atrophy (SMA) study.
In 24 treated patients aged 0.5 to 12 years—all of whom received at least two doses of salanersen—the investigational therapy led to an approximately 75% decrease in neurofilament light chain (NfL) concentration at six months. Biogen called this effect “meaningful,” adding that it was sustained through at least one year of follow-up, according to a Wednesday press release.
These results support salanersen’s potential success in Phase 3 development, Jefferies analysts told investors in a Wednesday note.
Twelve of the 24 treated patients also hit at least one new motor milestone after starting salanersen treatment. All participants were able to maintain their baseline motor skills throughout the study.
Biogen will move forward with an 80-mg dose of salanersen and has planned a global Phase 3 program to assess a once-yearly dose across a broad population of patients with SMA. There will be three studies: STELLAR-1, which will focus on infants under six weeks old, STELLAR-2, which will administer salanersen around six months after a gene therapy infusion in infants and SOLAR, which is an open-label study of patients 15 to 60 years of age.
All three studies are slated to begin this year.
Edgewise Touts Functional Benefits in Becker Muscular Dystrophy
Edgewise Therapeutics presented long-term data on its drug candidate sevasemten for Becker muscular dystrophy (BMD), a rare genetic neuromuscular condition that leads to progressive and irreversible muscle loss.
The biotech had previously run the ARCH and CANYON trials, participants from which were allowed to roll into the open-label MESA study, which followed their long-term progress. Open-label extension data presented at MDA showed that sevasemten sustained stability outcomes through 3.5 and 2 years of follow-up in ARCH and CANYON, respectively.
MESA used the North Star Ambulatory Assessment to measure functional performance in patients and additionally found that scores of those treated with sevasemten “diverged markedly” from natural history controls, in whom performance deteriorated, according to Edgewise’s news release on Tuesday.
“We believe the data continue to support sevasemten’s efficacy benefit in BMD, for which there are no approved therapies,” analysts at Truist Securities wrote in a Wednesday note.
Edgewise is currently running the pivotal, placebo-controlled GRAND CANYON trial for sevasemten, with topline data expected in the fourth quarter. If positive, the biotech will work toward an approval package for the drug.
Sarepta’s DMD Data Further Build Product Profiles
Sarepta Therapeutics presented a post-hoc analysis of the confirmatory ESSENCE study at MDA, showing that its exon-skippers for Duchenne muscular dystrophy, Vyondys 53 and Amondys 45, delayed the time to motor decline by around 23 weeks versus placebo, according to a poster at the conference.
The two phosphorodiamidate morpholino oligomers (PMOs) are currently cleared for marketing through an FDA accelerated approval, meaning the company still has to validate their clinical benefit in a Phase 3 confirmatory study.
In November 2025, the drugs failed that study, called ESSENCE, unable to significantly improve motor function versus placebo. Sarepta nevertheless said at the time that it would pursue traditional approval of the pair. The company is set to meet with the FDA in March to make its case for Vyondys and Amondys, but with ESSENCE’s failure, the regulator has the authority to pull the drugs from the market.
Outcomes of the post-hoc analysis should “support FDA discussions in March 2026 on a path forward for full approval,” Jefferies analysts said in a note on Wednesday.
Also at the meeting, Sarepta presented 3-year data from the EMBARK study of the gene therapy Elevidys. Results support Elevidys’ therapeutic benefit in ambulatory patients at 2-3 years of follow-up, showing deepening functional and cardiac benefits, especially as compared with external, untreated controls, according to Jefferies.
Elevidys was at the center of a safety controversy last year after being linked to two deaths, both in non-ambulatory patients. The therapy has since been slapped with a boxed warning and has had its patient pool narrowed.
