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 &...
by Peter Ciszewski | Oct 15, 2019
Neena Nizar, PhD, founder of the Jansen’s Foundation, is the personification of the determination. Born with a rare disease that was not properly diagnosed until after her sons’ were also diagnosed with the condition (Jansen’s metaphyseal...
