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...
by Peter Ciszewski | Jun 30, 2021
William D. Tap, MD, Chief of the Sarcoma Medical Oncology Service at Memorial Sloan Kettering Cancer Center, gives an overview of tenosynovial giant cell tumors (TGCT). As Dr. Tap explains, TGCT are a group of rare, benign tumors that involve the synovium,...
