Noah Stansfield

Barry J Byrne, MD, PhD, the chief medical advisor of MDA, also spoke about what he personally is most looking forward to at this year's meeting.

MARINA trial shows del-desiran siRNA reaches muscle, lowers toxic DMPK RNA in DM1, with early functional gains.

Two-year phase 3 data show Elevidys gene therapy slows Duchenne decline in boys, improving NSAA and walk tests with manageable safety.

The chief medical advisor at the Muscular Dystrophy Association also discussed what he personally is looking forward to at the conference this year. [WATCH TIME: 4 minutes]

52-week trial data shows inebilizumab sustains gains in generalized myasthenia gravis, cuts prednisone, and spotlights CD

In honor of Duchenne Muscular Dystrophy Awareness Week, observed February 13 to 19, we're taking a look at the pipeline of therapeutic candidates in development for DMD.

Phase 2 MoonStone trial shows obexelimab sharply cuts new MRI lesions in relapsing MS by week 12, with convenient, weekly self-injection.

The FDA issued a CRL for gene therapy RGX-121 in Hunter syndrome, citing trial design and biomarker doubts; Regenxbio seeks a meeting with the agency and resubmission.

Ultragenyx seeks FDA accelerated approval for UX111 gene therapy in Sanfilippo syndrome, citing neurologic benefit and the use of cerebral spinal fluid heparan sulfate data.

The analysis included 280 patients from CLARIFY-MS extension and 219 patients from MAGNIFY-MS extension, a total of 499 patients with at least 4 years of follow-up.

Specifically, the alignment relates to a separate, planned clinical trial referred to as ASPIRE.

With regard to safety, there were no serious adverse events (AEs) or AEs of special interest deemed related to OCU410ST reported in the study.

Intellia’s trial for nex-z in ATTR-CM remains under clinical hold.

The associate chief medical officer at Michigan State University highlighted advances in neuromuscular disease, emerging therapeutic strategies, and the growing role of patient-reported outcomes at AANEM 2025. [WATCH TIME: 2 minutes]

The consultant neurologist at the National Neuroscience Institute discussed how artificial intelligence, biomarker development, and drug repurposing can help improve patient care in ALS. [WATCH TIME: 4 minutes]

AskBio noted that AB-1009 also recently received fast track and orphan drug designations from the FDA.

The therapy also received orphan drug designation from the FDA.

In the middle of 2026, the company is planning to submit a BLA for RGX-202 via an accelerated approval pathway.

At NSGC 2025, the research director at the Clinic for Special Children discussed the clinic’s approach to accelerating the diagnosis and treatment of spinal muscular atrophy. [WATCH TIME: 5 minutes]

The senior director of research and real world data at Genome Medical talked about the benefits and challenges of genetic counselors adopting artificial intelligence tools. [WATCH TIME: 5 minutes]

The genetic counselor in the Department of Ophthalmology at the University of Pittsburgh Medical Center talked about the evolving role of genetic counselors in guiding patients through gene therapy. [WATCH TIME: 4 minutes]

The associate professor of physical medicine and rehabilitation at Virginia Commonwealth University discussed his passion for educating others about electrodiagnosis and ultrasound in neuromuscular disorders.

The neuroscience program coordinator at Loyola University Medical Center talked about a recent global nurse survey for those caring for patients with generalized myasthenia gravis. [WATCH TIME: 5 minutes]

The associate professor of clinical neurology at Keck Medicine of USC discussed advancements in therapeutics, the growing focus on biomarkers, and ongoing challenges in myasthenia gravis. [WATCH TIME: 4 minutes]

The associate professor of neurology at Yale School of Medicine discussed 52-week data from the phase 3 MINT trial of inebilizumab in patients with generalized myasthenia gravis. [WATCH TIME: 5 minutes]

The head of Rare Diseases US at UCB talked about the company’s latest data on rozanolixizumab and zilucoplan in patients living with generalized myasthenia gravis. [WATCH TIME: 3 minutes]

The chief medical advisor at the Muscular Dystrophy Association shared key highlights of what attendees can expect at the 2025 MDA Clinical and Scientific Conference. [WATCH TIME: 4 minutes]

According to recent studies and the latest insights provided by experts, early developments in gene and cell therapies show promise for patients living with Parkinson disease, but challenges remain.

Following a successful first patient treatment experience with TSHA-102, an adeno-associated virus vector-based gene therapy for Rett syndrome, Taysha Gene Therapies was recommended to continue with investigating the therapy in a second patient.