by Madaline Spencer | Mar 26, 2025
Takayuki Shirakawa, PhD, Research Scientist at Mitsubishi Tanabe Pharma, discusses the effects of LRSAM1 on TDP-43 in patients with amyotrophic lateral sclerosis (ALS). ALS, also referred to as “Lou Gehrig’s disease,” is a motor neuron disease...
by Madaline Spencer | Mar 24, 2025
Alessandra Maleddu, MD, Sarcoma Specialist at the University of Colorado, discusses the misdiagnosis of desmoid tumors and provides some resources for patients. Desmoid tumors are rare, benign, locally invasive, soft tissue tumors. They are commonly...
by Madaline Spencer | Mar 21, 2025
Kelly Guillo, Board Member of the Prader-Willi Syndrome Association in Georgia, discusses Prader-Willi syndrome (PWS) from the perspective of a caregiver. PWS is a rare genetic condition that affects many parts of the body but its most dominant symptom...
by Madaline Spencer | Mar 20, 2025
Bruce Leuchter, MD, Co-founder and CEO of Neurvati Neurosciences and GRIN Therapeutics, discusses the Breakthrough Therapy designation for radiprodil for treatment of patients with GRIN-related neurodevelopmental disorders. GRIN-related...
by Madaline Spencer | Mar 19, 2025
Steven Pipe, MD, Professor of Pediatrics and Pathology at the University of Michigan, discusses positive follow-up results in patients with hemophilia B treated with Hemgenix (etranacogene dezaparvovec-drlb). Hemophilia B is a rare bleeding disorder that...
