by Madaline Spencer | Aug 29, 2025
The U.S. Food and Drug Administration (FDA) has approved Papzimeos (zopapogene imadenovec-drba) for the treatment of adults with recurrent respiratory papillomatosis (RRP). RRP is a rare viral disease characterized by benign tumors in the respiratory tract. The tumors...
by Madaline Spencer | Aug 29, 2025
Tracey Dawson, PhD, SVP, U.S. Therapeutic Area Head of Neuroscience at Novartis, discusses the SMAshing My Limits campaign. Spinal muscular atrophy (SMA) is a group of genetic neuromuscular disorders that affect the motor neurons. The loss of motor...
by Madaline Spencer | Aug 28, 2025
Mario Maldonado, MD, Global Head of Clinical Development for Endocrinology at Recordati Rare Diseases, discusses Recordati’s presentations at ENDO 2025. LINC6 Clinical Trial LINC 6 is an ongoing, prospective observational study evaluating the long-term...
by Madaline Spencer | Aug 27, 2025
Alan Krasner, MD, Chief Endocrinologist at Crinetics Pharmaceuticals, discusses atumelnant for the treatment of patients with congenital adrenal hyperplasia (CAH). CAH is a genetic condition characterized by an enzyme deficiency in the adrenal glands....
by Madaline Spencer | Aug 26, 2025
Jason Crompton, PharmD, Global Medical Director of the Acromegaly Program at Chiesi Rare Diseases, discusses disparities between biochemical control and symptom burden in patients with acromegaly. Acromegaly is a hormonal disorder that results from the...
