Muscular Dystrophy Association

The chief scientific officer at Precision Biosciences discussed the durable potential of ARCUS gene editing and the company’s next steps toward in-human trials for Duchenne muscular dystrophy.

The chief scientific officer at Precision Biosciences discussed the ARCUS gene editing platform and its potential application in Duchenne muscular dystrophy through a durable, AAV-delivered therapeutic strategy.

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.

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.

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.

Barry J Byrne, MD, PhD, the chief medical advisor of MDA, also shared his thoughts on the 75th anniversary of the organization.

Barry J Byrne, MD, PhD, the chief medical advisor of MDA, discussed what attendees can expect at the 2025 MDA Meeting.

The chief medical advisor of the Muscular Dystrophy Association also shared his thoughts on the 75th anniversary of the organization.

The chief medical advisor of the Muscular Dystrophy Association discussed what attendees can expect at the 2025 MDA Meeting.

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

The data comes from 7 patients treated in the phase 1/2 IGNITE-DMD study (NCT03368742).

The real-world data, which includes patients who received Zolgensma before and after tracheostomy, comes from the noninterventional RESTORE registry (NCT04174157).

In observance of Rare Disease Day 2024, the vice president of public policy and advocacy at the Muscular Dystrophy Association discussed advocacy and policy progress regarding rare neuromuscular diseases.

Barry J Byrne, MD, PhD, the chief medical advisor of MDA, discussed what he views as the next horizon in the field of gene therapy.

Barry J Byrne, MD, PhD, the chief medical advisor of MDA, discussed his research on a new application of efgartigimod alfa (Vyvgart).

Barry J Byrne, MD, PhD, the chief medical advisor of MDA, discussed MDA Care Centers and his excitement for the organization’s the Upcoming Clinical & Scientific Conference.

The chief medical advisor of the Muscular Dystrophy Association discussed how directed evolution may help develop AAV capsids better suited for treating neuromuscular diseases.

The chief medical advisor of the Muscular Dystrophy Association discussed research he is working on with the goal of increasing eligibility for gene therapies and opening the door to redosing.

The chief medical advisor of the Muscular Dystrophy Association discussed what seasoned clinicians and newcomers to the field alike can look forward to at the 2024 MDA Meeting.

Sharon Hesterlee, PhD, the chief research officer of MDA, discussed highlights of 2023’s meeting and exciting new things to look forward to at the upcoming 2024 meeting.