by Peter Ciszewski | Jul 8, 2021
Heather Olson, MD, MS, Neurologist at Boston Children’s and Neurology Instructor at Harvard Medical, discusses FOXG1 Research Foundation’s Natural History Study. FOXG1 syndrome is a neurological condition characterized by impaired development and...
by Peter Ciszewski | Jul 7, 2021
Tim Miller, PhD, CEO, President, and Co-Founder of Forge Biologics, discusses the phase 1/2 RESKUE study which will evaluate FBX-101 for the treatment of Krabbe disease. This clinical trial is currently recruiting. Krabbe disease is a neurodegenerative...
by Peter Ciszewski | Jul 6, 2021
Tim Miller, PhD, CEO, President, and Co-Founder of Forge Biologics, gives an overview of Krabbe disease. As Dr. Miller explains, Krabbe disease is a neurodegenerative disorder caused by a deficiency of galactosylceramidase. Deficiency of this enzyme impairs...
by Peter Ciszewski | Jul 5, 2021
Adrian Hepner, MD, PhD, Chief Medical Officer at Pharnext, discusses how patients with Charcot-Marie-Tooth disease type 1A (CMT1A) are usually diagnosed. CMT1A is a rare inherited neurological disorder that causes damage to the peripheral nerves. It is caused...
by Peter Ciszewski | Jul 1, 2021
Adrian Hepner, MD, PhD, Chief Medical Officer at Pharnext, discusses the history of PXT3003, an investigational combination therapy for the treatment of Charcot-Marie-Tooth disease Type 1A (CMT1A). CMT1A is a rare inherited neurological disorder that causes...
