by Madaline Spencer | Mar 21, 2023
An HoFH patient describes his diagnostic journey following years of symptoms. Peter, a resident of San Diego, was diagnosed with homozygous familial hypercholesterolemia (HoFH) at age 27 by an alert urgent care physician. In hindsight, Peter had displayed...
by Madaline Spencer | Mar 17, 2023
Arthur Suckow, PhD., chief executive officer at DTx Pharma talks about Charcot-Marie-Tooth Disease Type 1A (CMT1A) and a treatment in development for this rare disease. CMT1A is a rare inherited neurological disorder that causes damage to the peripheral nerves. It is...
by Madaline Spencer | Mar 11, 2023
Art Levin, PhD, distinguished scientist and strategic leader and member of the company’s board of directors at Avidity Biosciences, discusses positive data from the preliminary assessment of the phase 1/2 MARINA trial of AOC 1001 for treatment in patients with...
by Madaline Spencer | Mar 10, 2023
Art Levin, PhD, distinguished scientist and strategic leader and member of the company’s board of directors at Avidity Biosciences, at Avidity Biosciences, gives an overview of myotonic dystrophy type 1 (DM1). As explained by Dr. Levin, myotonic dystrophy type...
by Madaline Spencer | Mar 9, 2023
Kyle Wood, MD, Associate Professor of Urology, University of Alabama at Birmingham, discusses the results of the phase 1b study evaluating SYNB8802 for the treatment of enteric hyperoxaluria. Enteric hyperoxaluria is a rare metabolic disorder characterized by...
