by Peter Ciszewski | Dec 4, 2021
Ashish Pradhan, MD, Executive Director and Disease Area Lead for MS and NMOSD at Genentech, discusses the 4-year data examining the efficacy and safety of satralizumab to treat neuromyelitis optica spectrum disorder (NMOSD). This data was recently presented at...
by Peter Ciszewski | Dec 3, 2021
Omar Sinno, MD, Medical Lead of Rare Diseases at UCB, describes a recent publication about the real-life impact of myasthenia gravis (MG). Myasthenia gravis is a chronic autoimmune neuromuscular disease characterized by weakness of the skeletal muscles. Common...
by Peter Ciszewski | Dec 2, 2021
Kevin and Amy Young, parents of a young boy with KAT6A syndrome, discuss the current management strategies for this rare disease. KAT6A syndrome is an ultra-rare genetic condition caused by a mutation in the KAT6A gene. Common symptoms may include developmental...
by Peter Ciszewski | Dec 2, 2021
Jeroen Valkenburg, General Manager of the Lumasiran program at Alnylam Pharmaceuticals, describes the results of ILLUMINATE-C, a phase 3 clinical trial evaluating the safety and efficacy of lumasiran in advanced primary hyperoxaluria type 1 (PH1) patients. PH1...
by Peter Ciszewski | Dec 2, 2021
The U.S. Food and Drug Administration (FDA) has granted accelerated approval to vosoritide (Voxzogo), indicated to increase linear growth in pediatric patients 5 years of age or older with achondroplasia with open epiphyses. Achondroplasia is a skeletal dysplasia...
