by Madaline Spencer | Mar 18, 2025
Alan Percy, MD, Pediatric Neurologist at the University of Alabama at Birmingham, discusses results from the DAFFODIL study evaluating long-term safety of trofinetide in girls ages two to four years with Rett syndrome. Rett syndrome is a...
by Madaline Spencer | Mar 17, 2025
Reena Sharma, MD, Adult Metabolic Consultant at Salford Royal Hospital and University of Manchester, discusses plans for a phase 3 clinical trial evaluating FLT201 in patients with Gaucher disease. Gaucher disease refers to a rare group of inherited metabolic...
by Madaline Spencer | Mar 14, 2025
Majdolen Joleen Istaiti, Clinical Study Coordinator, Gaucher Unit at Shaare Zedek Medical Center, discusses insights into the cancer risk associated with Gaucher disease. Gaucher disease is a rare lysosomal disorder that leads to the harmful accumulation...
by Madaline Spencer | Mar 13, 2025
Caroline Hastings, MD, Professor of Pediatrics at UCSF Benioff Children’s Hospital Oakland, discusses an open-label study of patients under 3 years of age with Niemann-Pick disease type C (NPC) being treated with Trappsol Cyclo (HPβCD). NPC is a...
by Madaline Spencer | Mar 13, 2025
The U.S. Food and Drug Administration has approved Encelto (revakinagene taroretcel-lwey) for the treatment of macular telangiectasia type 2 (MacTel). MacTel is a rare neurodegenerative disease characterized by localized retinal degeneration. Patients with this...
