by Peter Ciszewski | Jun 8, 2021
Meghann Ferguson, Director-at-Large at the National Niemann-Pick Disease Foundation and mother of a 7-year-old boy with Niemann-Pick Disease Type C (NPC), describes her son’s diagnostic journey. NPC is a disabling neurogenetic disorder that has been diagnosed...
by Peter Ciszewski | Jun 3, 2021
Rebecca Aune, Director of Education Programs for the National Organization for Rare Disorders (NORD), talks about the upcoming 2021 Living Rare, Living Stronger NORD Patient and Family Forum being held June 26-27. The event will be held virtually. As Ms. Aune...
by Peter Ciszewski | Jun 2, 2021
Quinn Dinh, MD, Vice President of Medical Affairs at Horizon Therapeutics, discusses what clinicians ought to know about neuromyelitis optica spectrum disorder (NMOSD). NMOSD is a rare central nervous disorder that primarily affects the spinal cord and optic...
by Peter Ciszewski | Jun 1, 2021
Julia Jenkins, Executive Director at Everylife Foundation, describes three of the organization’s goals for newborn screening. As Ms. Jenkins explains, the primary goal is to pass legislation in each state that requires newborn screening for all diseases on the...
by Peter Ciszewski | May 31, 2021
Meghann Ferguson, Director-at-Large at the National Niemann-Pick Disease Foundation and mother of a 7-year-old boy with Niemann-Pick Disease Type C (NPC), talks about the current crisis NPC families are facing as adrabetadex, an experimental drug being...
