Innovations in Science

Stathmin-2 loss a new hallmark forTDP-43-dependent Amyotrophic Lateral Sclerosis
Jone Lopez-Erauskin, PhD (Chair)

VCP inhibitor CB-5083: a potential treatment for VCP inclusion Body Myopathy
Virginia Kimonis, MD, MRCP (Chair)

Inhibition of PKC alleviates nucleocytoplasmic transport disruption in FUS-mediated ALS
Clotilde Lagier-Tourenne, MD, PhD

“Designer DNA drug” therapies for sporadic ALS: restoration of stathmin-2oruse of “identity theft” to generate replacement neurons
Don Cleveland, PhD

Update on Rare Disease Programs at Denali
Joseph Lewcock, PhD

Session Moderator
R. Rodney Howell, MD (Chair)

Newborn Screening for Neuromuscular Conditions
Brittany Hernandez

Newborn Screening: How to Prepare Your MDA Care Center
Marcia Felker, MD

Newborn Screening for SMA: Wisconsin Experience
Mei Baker, MD

Session Moderator
Merit Cudkowicz, MD (Chair)

Session Moderator
Richard Finkel, MD (Chair)

Biostatistical considerations in clinical trial design in neuromuscular disorders
Ken Cheung, PhD

Clinical considerations for efficient design of clinical trials for neuromuscular diseases
Kevin Flanigan, MD

“Common” neuromuscular diseases, with large-scale studies, using ALS as an example
Sabrina Paganoni, MD

Rare neuromuscular disease considerations, using giant axonal neuropathy and myotubular myopathy as examples
Diana Bharucha-Goebel, MD

Virtual Industry Forum: Presented by Sarepta Therapeutics
Natural History and Therapeutic Options in Development for DMD
Craig McDonald, MD
Crystal Proud, MD
Louise Rodino-Klapac, PhD

Jahannaz Dastgir, DO