by Madaline Spencer | Oct 9, 2026
Michael Benatar, MD, PhD, Department of Neurology and ALS Center, University of Miami Miller School of Medicine, discusses the use of plasma proteomics in predicting clinical manifestations of amyotrophic lateral sclerosis (ALS). ALS is a rare motor neuron...
by Madaline Spencer | Oct 8, 2026
Lukasz Jarzyna, Vice President of Rare Disease at LEO Pharma, discusses the Priority Review for dersimelagon in the treatment of erythropoietic protoporphyria (EPP) and X-linked protoporphyria (XLP). EPP and XLP are rare congenital metabolic disorders...
by Madaline Spencer | Oct 6, 2026
The US Food and Drug Administration (FDA) has approved Camzyos (mavacamten) for the treatment of symptomatic obstructive hypertrophic cardiomyopathy (oHCM) to improve functional capacity and symptoms in patients weighing 30 kg (66 lbs) or more. oHCM is a rare genetic...
by Madaline Spencer and Ailene Cantelmi | Oct 5, 2026
Paz Vellanki, MD, PhD, Vice President of Clinical Development at Precision for Medicine, discusses the FDA CDER’s Accelerating Rare Disease Cures (ARC) Program. The ARC Program was launched in 2022 with the goal of accelerating the development of safe and...
by Madaline Spencer | Oct 1, 2026
The US Food and Drug Administration (FDA) has approved Gazyva (obinutuzumab) to reduce the risk of relapse in adult and pediatric patients 2 years of age and older with frequently relapsing or steroid dependent childhood-onset idiopathic nephrotic syndrome (INS) who...
