by Peter Ciszewski | Oct 21, 2019
Joe Pugliese of the Hemophilia Alliance, a not-for-profit organization of federally funded hemophilia treatment centers that is working to ensure its member have the expertise, resources and public support to sustain their integrated clinical and pharmacy...
by Peter Ciszewski | Oct 20, 2019
The Global Commission to End the Diagnostic Odyssey for Children, co-chaired by Takeda, Microsoft and EURORDIS-Rare Diseases Europe, is a multidisciplinary group of patient advocates, physicians and other experts working together to help solve the complex...
by Peter Ciszewski | Oct 17, 2019
In less than one month, Disorder: The Rare Disease Film Festival will be underway. The only film festival that focuses exclusively on rare diseases will take place November 9-10, 2019 at the Mission Bay Conference Center in San Francisco. The two-day event will...
by Peter Ciszewski | Oct 16, 2019
The U.S. Food and Drug Administration (FDA) granted approval to Scenesse (afamelanotide) to increase pain-free light exposure in adult patients with a history of phototoxic reactions (damage to skin) from erythropoietic protoporphyria (EPP). For patients who are...
by Peter Ciszewski | Oct 16, 2019
Luke Rosen is the father of a daughter with KIF1A associated neurological disorder (KAND) and co-founder of the highly successful patient organization, KIF1A.org. Rosen also works with Ovid Therapeutics as their Vice President of Patient Engagement &...
