by Peter Ciszewski | Oct 29, 2021
Gary Joseph Lelli, MD, of the Department of Ophthalmology at Weill Cornell Medicine, describes the thyroid eye disease (TED) patient population. TED is a rare autoimmune disease causing permanent facial disfigurement which can severely affect patients’ quality...
by Peter Ciszewski | Oct 28, 2021
Results from several clinical trials of SRP-9001 show the investigative gene therapy induces sustained functional improvements in people with Duchenne muscular dystrophy (DMD). While presenting these data, Sarepta (the manufacturer) also offered more details about...
by Peter Ciszewski | Oct 28, 2021
Matthew Klein, MD, MS, FACS, Chief Development Officer at PTC Therapeutics, discusses PTC-923, an investigational cofactor therapy for phenylketonuria (PKU). PKU is a rare genetic metabolic disorder that results in reduced activity of phenylalanine hydroxylase...
by Peter Ciszewski | Oct 27, 2021
Jennifer Gudeman, PharmD, Vice President of Medical and Clinical Affairs at Avadel Pharmaceuticals, discusses the American Association of Sleep Medicine’s (AASM) updated clinical practice guidelines for narcolepsy. Narcolepsy is a rare neurological disorder...
by Peter Ciszewski | Oct 26, 2021
Avanish Vellanki, Cofounder and CEO at Rain Therapeutics, discusses the role of p53 and MDM2 in cancers like liposarcoma. As Mr. Vellanki explains, p53 regulates the cell cycle, and in that role is essential for tumor suppression. MDM2 is a crucial regulator of...
