by Peter Ciszewski | Apr 8, 2019
The U.S. Food and Drug Administration (FDA) granted Fast Track designation to ABO-101, a gene therapy for Sanfilippo syndrome type B (MPS IIIB). ABO-101 is designed to deliver a functional copy of the NAGLU gene to the central nervous system and peripheral tissues....
by Peter Ciszewski | Apr 8, 2019
Becky Abbott, MPH, Manager of Treatment and Research at the National Foundation for Ectodermal Dysplasias (NFED) discuses why she attends Rare Disease Week. Ectodermal dysplasias (ED), a group of more than 180 disorders that affect the outer layer of tissue of the...
by Peter Ciszewski | Apr 8, 2019
Morie Gertz, MD, Hematologist and Chair of Internal Medicine at Mayo Clinic discussed the current unmet needs in the treatment of hereditary TTR amyloidosis, antisense technology, inotersen, and the NEURO-TTR study. Patients with hATTR-PN primarily experience...
by Peter Ciszewski | Apr 4, 2019
Raj Mehra, PhD, Chief Executive Officer of Seelos Therapeutics provides an overview of Sanfilippo syndrome, also known as Mucopolysaccharidosis type III (MPS III), a progressive disorder that primarily affects the brain and spinal cord (central nervous...
by Peter Ciszewski | Apr 4, 2019
Annette Bakker, President and Chief Scientific Officer of the Children’s Tumor Foundation, discusses neurofibromatosis (NF), a genetic disorder that affects 1 in every 3,000 people. There are three types of NF: NF1, NF2, and schwannomatosis. Neurofibromatosis...
