Gather your friends and family and join is for another invigorating 2-mile walk with beautiful views of Long Island Sound around Tod’s Point in Old Greenwich, and help us raise much needed funds to find a cure for Niemann-Pick type C disease. No beach pass, no problem. DART has you covered! The morning always makes …
Category
Showing: 4 - 6 of 7 RESULTS
Banner News
FDA Approves 2nd New Drug to Treat NPC
FDA Approves New Drug to Treat Niemann-Pick Disease, Type C | FDA For Immediate Release:September 24, 2024 Today, the U.S. Food and Drug Administration approved Aqneursa (levacetylleucine) for the treatment of neurological symptoms associated with Niemann-Pick disease type C (NPC) in adults and pediatric patients weighing at least 15 kilograms. “This is the second treatment …
FDA Approves 1st Treatment for NPC
FDA Approves First Treatment for Niemann-Pick Disease, Type C | FDA For Immediate Release:September 20, 2024 Today, the U.S. Food and Drug Administration approved Miplyffa (arimoclomol), an oral medication for the treatment of Niemann-Pick disease, type C (NPC). Miplyffa, in combination with the enzyme inhibitor miglustat, is approved to treat neurological symptoms associated with NPC in …