by Peter Ciszewski | Nov 23, 2017
Melissa P. Wasserstein, MD, Chief of Pediatric Genetic Medicine at the Children’s Hospital at Montefiore, New York City discusses three rare diseases that she sees at her clinic: Neimann Pick Disease, Krabbe Disease, and Chromosome 22q11.2 deletion syndrome...
by Peter Ciszewski | Nov 23, 2017
Len Walt, Vice President, Head of Medical Affairs, Sobi in North America, provides an overview of Still’s disease, a rare inflammatory disorder that affects the entire body, and how these patients typically present to their physicians. Still’s disease is...
by Peter Ciszewski | Nov 20, 2017
PellePharm, a clinical-stage biopharmaceutical company focusing on rare genetic dermatological conditions, announced that the Food and Drug Administration (FDA) has granted both Breakthrough Therapy Designation and Orphan Drug Designation to topical patidegib for...
by Peter Ciszewski | Nov 20, 2017
Jessica Fein’s daughter Dalia has the mitochondrial disease MERRF Syndrome. Jessica spoke with us at the Rare Disease Film Festival, an event showcasing a myriad of films from around the world that addresses the challenges of life with a rare disease. Myoclonic...
by Peter Ciszewski | Nov 19, 2017
Philippe Maitre, Executive Vice President, US Operations for Onxeo discusses Livatag, a nanoparticle formulation of doxorubicin (Transdrug technology) developed for the treatment of primary liver cancer.
