A team of allergists, immunologists, and dermatologists from Germany and the United States sought to test a new patient-reported outcomes measure for assessing mastocytosis disease control.3 weeks ago
Drs. Patrick McKiernan and Nadia Ovchinsky discuss the recently published guidance on best practices to diagnose, treat, and monitor patients with progressive familial intrahepatic cholestasis (PFIC).2 months ago
This educational webinar on fibrodysplasia ossificans progressiva (FOP), hosted by Ellen Elias, MD and Christiaan Scott, MD, examines best practices to suspect and diagnose this ultra-rare condition.2 months ago
Greg Yanik, MD, Clinical Professor at University of Michigan's C.S. Mott Children's Hospital, provides an overview of this rare childhood cancer, goals of therapy, using the Curie score, and immunotherapy treatments.2 months ago
Rett syndrome thought leaders discuss how DAYBUE™ (trofinetide), the first and only FDA-approved treatment for Rett syndrome in adults and pediatric patients 2 years of age and older, plays a role in their patients’ treatment plans, while shedding light on their personal experiences with DAYBUE in their practices.5 months ago
This panel discussion by three clinical research leaders in HAE, Drs. Aleena Banerji, Timothy Craig, and Marc Riedl, provide an overview of the discrepancies in care observed in certain patient populations, as well as a discussion on best practices to reduce those inequalities moving forward.5 months ago
Hypoparathyroidism is a rare endocrine disorder characterized by a deficiency of parathyroid hormone (PTH), which leads to decreased serum calcium and increased phosphorus levels in the blood.5 months ago
Almost half of all novel medications approved by the U.S. Food and Drug Administration (FDA) are orphan drugs. Below is the list of important regulatory dates for all orphan drugs for 2025.6 months ago
Dr. Sujit Sheth discusses his recently published work in Blood Advances titled “Rates of severe neutropenia and infection risk in patients treated with deferiprone: 28 years of data."8 months ago
Type 1 galactosemia is a rare genetic disease that can be life-threatening in newborns and can lead to lifelong cognitive, neurological, and speech complications, as well as primary ovarian insufficiency in girls and women.8 months ago
Dr. Mark J. Buchfuhrer discusses clinical practice guidelines update suggesting against the standard use of dopamine agonists for the treatment of restless legs syndrome.8 months ago
Dr. Sonal Kumar, an Assistant Professor of Medicine, and Director of Clinical Hepatology at Weill Cornell Medical College in New York, NY discusses the integral role of the patient voice in navigating disease progression and determining an individualized care plan for people living with primary biliary cholangitis (PBC) that reflects their needs and treatment goals.8 months ago
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