Muscular Dystrophy Association Clinical and Scientific Conference (MDA)

Patients with Duchenne muscular dystrophy between ages of 6 and 24 months old demonstrated a similar safety profile on eteplirsen than those between 24 and 48 months of age.

In a real-world study, eterplirsen was safe for patients with Duchenne muscular dystrophy, with sustained or improved status in function.

Tadalafil shows potential in addressing microvascular impairment in Duchenne muscular dystrophy, with post-contractile MRI as a screening tool.

Jeff Chamberlain, PhD, a leading professor in gene therapy focused on Duchenne muscular dystrophy, shared his reaction to being named the recipient of the 2024 MDA Legacy Award, as well as the state of the DMD field currently.

The chief medical advisor at the Muscular Dystrophy Association provided perspective on the upcoming meeting and the conversations surrounding new therapeutics for diseases that once had little to nothing. [WATCH TIME: 4 minutes]

The professor in the Department of Translational Neuroscience at Barrow Neurological Institute provided perspective on the approval of tofersen (Qalsody; Biogen) for patients with ALS and the ways neurofilament light will be used going forward. [WATCH TIME: 3 minutes]

The professor in the Department of Translational Neuroscience at Barrow Neurological Institute discussed several topics related to ALS research and the emerging biomarkers in recent years ahead of the 2024 MDA Clinical and Scientific Conference.

The professor in the Department of Translational Neuroscience at Barrow Neurological Institute provided comments on her session track at the upcoming 2024 MDA conference and some of the recent advancements in ALS research. [WATCH TIME: 4 minutes]

The chief medical officer of QurAlis talked about the potential of QRL-201 in patients with ALS from her talk presented at the 2023 MDA conference. [WATCH TIME: 4 minutes]

The Lillehei professor in stem cell and regenerative cardiovascular medicine at the University of Minnesota talked about the potential of investigating inducted pluripotent stem cells in neuromuscular diseases at MDA’s 2023 conference. [WATCH TIME: 5 minutes]

The neuromuscular physician at Virginia Commonwealth University talked about the promising future of gene therapy and access to resources in Duchenne muscular dystrophy. [WATCH TIME: 5 minutes]

Sitra Tauscher-Wisniewski, MD, vice president clinical development and analytics, Novartis Gene Therapies, talked about the new data findings from a study on gene therapy Zolgensma at the 2023 MDA conference.

At the 2023 MDA’s Clinical and Scientific conference, the neurology resident at Penn Medicine talked about the challenges of delivering intrathecal therapies and the importance of planning clinical resources for patients with ALS. [WATCH TIME: 3 minutes]

The co-founder and chief executive officer of MyoGene Bio talked about the advantages of gene editing over traditional gene therapy as a long term treatment for neuromuscular diseases at MDA’s 2023 conference. [WATCH TIME: 3 minutes]

The research physical therapist at Nationwide Children’s Hospital talked about the role of physical therapy in the changing landscape of care with neuromuscular diseases at the 2023 MDA conference. [WATCH TIME: 5 minutes]

The associate professor of neurology at Columbia University spoke at the 2023 MDA conference about the unmet needs of patients with ALS and the potential of new gene therapies. [WATCH TIME: 5 minutes]

At the 2023 MDA conference, a pediatric endocrinologist from Cincinnati Children’s Hospital talked about the unmet needs in treating bone health for patients with Duchenne muscular dystrophy. [WATCH TIME: 5 minutes]

The associate professor of neurology at the University of Texas Health Science Center at Houston talked about advances in the classification of inflammatory myopathies at the recently concluded 2023 MDA conference. [WATCH TIME: 4 minutes]

A group of experts in the care of patients with neuromuscular diseases—Douglas Sproule, MD, MSc; David Brumbaugh, MD; Sharon Hesterlee, PhD; Lauren Treat, MD; Donovan Lott, PT, PhD, CSCS—shared their perspectives on hot topics of treatment and management from the 2023 MDA conference.

At the 2023 MDA conference, the professor for human genetics and neurology at the University of Miami Miller School of Medicine talked about the role of genetics in neuromuscular diseases and potential therapies. [WATCH TIME: 5 minutes]

At the 2023 MDA conference, the scientific research portfolio director at Muscular Dystrophy Association detailed a session on collaborative research in ALS and ongoing efforts in the field. [WATCH TIME: 3 minutes]

The director of the Ann Kimball and John W. Johnson Center for Cellular Therapeutics at Houston Methodist Hospital spoke on about advances in ALS research and promising therapies on the horizon at the 2023 MDA conference. [WATCH TIME: 8 minutes]

The director of clinical outcomes and research development in neurology at Stanford University spoke about the paradigm shift in neuromuscular therapy interventions at the 2023 MDA conference. [WATCH TIME: 5 minutes]

The director of the center for gene therapy at Nationwide Children's Hospital talked about the challenges and opportunities of gene therapies for neuromuscular diseases at the 2023 MDA conference. [WATCH TIME: 4 minutes]

The senior statistical scientist and director of consulting at Berry Consultants talked about innovative clinical trial design in ALS research at the 2023 MDA conference. [WATCH TIME: 5 minutes]

At the 2023 MDA conference, the professor of neurology at the University of Minnesota talked about the current understanding and challenges of Limb Girdle muscular dystrophy from a clinical perspective. [WATCH TIME: 5 minutes]

The senior vice president of research at The ALS Association talked about the complexities of ALS and the importance of collaboration in research at the 2023 MDA conference. [WATCH TIME: 5 minutes]

Time to milestones such as invasive and noninvasive ventilation, speech-generating devices, and hospice care took significantly longer while on intravenous edaravone.

The research associate professor in the department of physical therapy at the University of Florida talked about physical therapy inventions for neuromuscular diseases at the 2023 MDA conference. [WATCH TIME: 4 minutes]

The child neurologist at the University of Colorado School of Medicine talked about her presentation on end of life care for patients with neuromuscular diseases at the 2023 MDA conference. [WATCH TIME: 5 minutes]