Marco Meglio

Phase 3 HOPE-3 data further support deramiocel’s potential to preserve both skeletal and cardiac function in Duchenne muscular dystrophy while slowing progression of myocardial fibrosis.

New interim ASGCT 2026 findings suggest investigational AAV9 gene therapy GS-100 may improve motor and cognitive outcomes in children with NGLY1 Deficiency, while informing dose optimization and immune management strategies.

New ASGCT 2026 data suggest Encoded Therapeutics’ investigational gene therapy ETX101 may meaningfully reduce seizures and alter developmental trajectory in children with SCN1A-positive Dravet syndrome.

New phase 2 DAWN data presented at ARVO 2026 showed sustained improvements in visual function and favorable safety with laru-zova in patients with X-linked retinitis pigmentosa.

Intellia has initiated a rolling FDA submission for lonvo-z after phase 3 data showed significant reductions in hereditary angioedema attacks and treatment burden with a single dose.

Investigators discuss interim phase 1/2 INSPIRE DUCHENNE data and the emerging biomarker and safety profile of SGT-003 in Duchenne muscular dystrophy.

Diana Castro, MD, highlights early phase 1b findings on salanersen and its potential to reshape treatment burden in spinal muscular atrophy.

Sharon Hesterlee, PhD, the president and CEO of the Muscular Dystrophy Association, discussed the upcoming conference and the evolving therapeutic landscape in neuromuscular disease.

Mind Moments, a podcast from our sister site Neurology Live, held an exclusive interview with Barry Byrne, MD, PhD.

AAV2-BDNF gene therapy shows promise in preventing neuronal loss and cognitive decline in early Alzheimer disease, according to trial results.

Mind Moments®, a podcast from our sister site NeurologyLive®, held an exclusive interview with Rajesh Pahwa, MD.

The clinical neurophysiologist at Children’s National and the professor of neurology at The Ohio State University Wexner Medical Center discussed treatment considerations for patients already living with SMA.

The clinical neurophysiologist at Children’s National and the professor of neurology at The Ohio State University Wexner Medical Center discussed treatment decisions in newly diagnosed SMA.

The clinical neurophysiologist at Children’s National and the professor of neurology at The Ohio State University Wexner Medical Center discussed the evolving SMA treatment landscape.

Emerging gene and cell therapies aim to slow Parkinson disease progression. Explore clinical updates on seven promising candidates in development.

Matthew Alexander, PhD, a neuromuscular expert, discussed the evolving landscape of targeted therapies in muscular dystrophies, from fibrosis and glycosylation to combination strategies with gene therapy.

During the 12-month study, Descartes-08 was deemed well-tolerated.

Matthew Wicklund, MD, a professor of neurology at the University of Texas Health Science Center San Antonio, discussed the genetic basis of oculopharyngeal muscular dystrophy, as well as the primary challenges in diagnosing and treating the condition.

Cassandra Gorsuch, PhD, chief scientific officer at Precision Biosciences, discussed the company’s ARCUS gene editing platform, which is being assessed for Duchenne muscular dystrophy.

The professor of neurology at the University of Texas Health Science Center San Antonio discussed discussed challenges of therapeutic development for OPMD.

The professor of neurology at the University of Texas Health Science Center San Antonio discussed the genetic basis of oculopharyngeal muscular dystrophy, as well as the primary challenges in diagnosing and treating the condition.

Donovan Decker, recipient of the 2025 MDA Legacy Award for Community Impact and Research, discussed his powerful journey as a patient advocate and gene therapy pioneer, shedding light on challenges and progress in LGMD.

Michael Flanagan, PhD, the chief scientific officer at Avidity, discussed the function and mechanism of the investigational antisense treatment, which is in development for DMD amenable to exon 44 skipping.

The chief scientific officer at Avidity Biosciences discussed the function and mechanism of the investigational antisense treatment, which is in development for DMD amenable to exon 44 skipping.

The findings were presented at the 2025 Muscular Dystrophy Association Clinical & Scientific Conference

Adverse events related to the gene therapy itself were mild or moderate, with most occurring in the first 90 days posttreatment.

John Brandsema, MD, a pediatric neurologist in the Division of Neurology at Children’s Hospital of Philadelphia, offered insights into the obstacles the clinical community is facing around integrating gene therapies into clinical practice.

The pediatric neurologist at Children’s Hospital of Philadelphia offered insights into the obstacles the clinical community is facing around integrating gene therapies into clinical practice.

Following up on Rare Disease Day, observed on February 28, Mary Schroth, MD, FAAP, FCCP, chief medical officer at Cure SMA, shared insights on the advances and promise for treating spinal muscular atrophy.