by Peter Ciszewski | Sep 9, 2019
Luke Rosen, father of a daughter with KIF1A associated neurological disorder (KAND), and co-founder of KIF1A.org talks about the signs and symptoms of KAND and how it is the “perfect storm’ of systems that has led him and other parents to start a advocacy group to...
by Peter Ciszewski | Aug 30, 2019
Jeffrey W Olin, DO is a Professor of Medicine at the Icahn School of Medicine at Mount Sinai and Director of Vascular Medicine and the Vascular Diagnostic Laboratory in the Zena and Michael A. Wiener Cardiovascular Institute and Marie-Josée and Henry R. Kravis...
by Peter Ciszewski | Aug 29, 2019
The Rare Disease Legislative Advocates (RDLA) program provides an opportunity for rare disease patients and advocates to share their information with policy makers and political leaders. They do this in a number of ways, including the scheduling of regular...
by Peter Ciszewski | Aug 28, 2019
Angelman syndrome is a rare neurodevelopmental disorder caused by a genetic mutation – usually a mutation in the ubiquitin protein ligase E3a (UBE3A) gene – that leads to a reduction in UBE3A protein. Symptoms include physical abnormalities (small head,...
by Peter Ciszewski | Aug 27, 2019
Orchard Therapeutics is a fairly new gene therapy company that has a pipeline more appropriate for a bigger, more established biotech. “People are surprised when they hear that actually Orchard has only been around for four years but we have one of the most...
