Muscular Dystrophy Association Clinical and Scientific Conference (MDA)

The former FDA commissioner discussed the necessity of policy and funding efforts for neuromuscular disorders, the stress on the clinical care system, and the need to acknowledge healthcare inequities.

The codirector of pediatric neuromuscular clinical research at Washington University in St. Louis discussed the session she chaired at MDA’s 2025 conference.

In pooled data from 156 patients, there were no deaths or study discontinuations.

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.

One of the patients, who was aged 3 years at the time of treatment, achieved 122.3% microdystrophin expression compared to control.

Patients treated at Dose 2 in the dose-escalation phase of Part 1 of the study showed a persistent 68% reduction in CK levels over 2 years.

OAV101 IT is a version of Zolgensma that is delivered directly to the spine.

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.

Sharon Hesterlee, PhD, the chief research officer of the Muscular Dystrophy Association discussed the upcoming MDA Clinical and Scientific Conference, which will be held March 16-19, in Dallas, Texas.

The chief research officer of the Muscular Dystrophy Association discussed the upcoming MDA Clinical and Scientific Conference, which will be held March 16-19, in Dallas, Texas.

Paul Melmeyer, MPP, the executive vice president of public policy & advocacy at MDA, discussed the growing interest in gene therapy for the annual meeting.

The vice president of Public Policy & Advocacy of the Muscular Dystrophy Association discussed the growing interest in gene therapy for attendees of the annual meeting.

In honor of Usher Syndrome Awareness Day, CGTLive® interviewed Zheng-Yi Chen, DPhil, associate professor, Otolaryngology–Head and Neck Surgery, Harvard Medical School, about the current state of research in this rare disease.

Jeffrey Chamberlain, PhD, McCaw Endowed Chair of Muscular Dystrophy at University of Washington, shared his outlook on the trajectory of research in the field.

Jeffrey Chamberlain, PhD, McCaw Endowed Chair of Muscular Dystrophy at University of Washington, highlighted studies presented at MDA's 2024 conference.

Jeffrey Chamberlain, PhD, McCaw Endowed Chair of Muscular Dystrophy at University of Washington, discussed his research career with muscular dystrophies.

The clinical professor of neurology and pediatrics at Keck School of Medicine of USC also discussed current strategies with gene therapy administration.

The lead scientist at Percheron Therapeutics discussed research on antisense oligonucleotide therapies in mouse models of DMD.

The McCaw Endowed Chair of Muscular Dystrophy at University of Washington shared his outlook on the trajectory of research in the field.

The clinical professor of neurology and pediatrics at Keck School of Medicine of USC discussed her talk on preparing for gene therapy administration.

Omer A. Abdul Hamid, MD, pediatric neurologist at Nemours Children’s Health offered advice and discussed important practical considerations with administering gene therapies.

The senior research scientist at RTI International discussed how patient preference studies can help patients with rare diseases have their voices heard.

The pediatric neurologist at Nemours Children’s Health offered advice and discussed his experiences delivering pediatric gene therapy.

The senior research scientist at RTI International discussed the progress that has taken place in recent years with regard to patient preference research.

The lead scientist at Percheron Therapeutics discussed a phase 2 blinded study of ATL1102 being conducted in the Europe and Australia.