by Peter Ciszewski | Jun 24, 2019
At the BIO International Convention recently held in Philadelphia, PA, we talked with Christina Hartman, Senior Director of Advocacy & Policy for the EveryLife Foundation for Rare Diseases about some of the efforts the non-profit organization is doing to...
by Peter Ciszewski | Jun 23, 2019
The FDA has granted orphan drug status and a rare pediatric disease designation to Denali Therapeutics pipeline candidate, DNL310. The pre-clinical candidate is being evaluated for the treatment of patients with mucopolysaccharidosis II (MPS II), also known as Hunter...
by Peter Ciszewski | Jun 21, 2019
Stephen Agyenim-Boateng, PharmD, RPH, is a medical consultant and board member of the non-profit organization, Sickle Cell 101. Sickle Cell 101 provides a global platform to provide educational content and resources for the patient population. They also conduct...
by Peter Ciszewski | Jun 20, 2019
Rare Disease International (RDI) is a global alliance of rare disease patient organizations designed to be an international voice for the rare disease community. One of its key initiatives is to include rare diseases in the Universal Health Coverage being...
by Peter Ciszewski | Jun 19, 2019
The U.S. Food and Drug Administration (FDA) has granted Fast Track designation to a novel one-time AAV9 gene therapy for CLN1 disease. The gene therapy is designed to deliver a functional copy of the PPT1 gene to the central nervous system and peripheral organs using...
