Trofinetide for the Treatment of Rett Syndrome
Ponni Subbiah, MD, MPH, Senior Vice President Global Head of Medical Affairs and Chief Medical Officer, Acadia Pharmaceuticals, discusses trofinetide for the treatment of Rett syndrome.
Ponni Subbiah, MD, MPH, Senior Vice President Global Head of Medical Affairs and Chief Medical Officer, Acadia Pharmaceuticals, discusses trofinetide for the treatment of Rett syndrome.
SACRAMENTO, CA, April 24, 2024—Today, California is one step closer to establishing a Rare Disease Advisory Council (RDAC) to help the 1-in-10 Californians
This very special event will celebrate NORD’s legacy and achievements, and honor those individuals and organizations doing remarkable work on behalf of the rare disease…
Priya Kishnani, MD, Professor of Pediatrics at Duke University Medical Center, discusses intrauterine treatment for lysosomal diseases.
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Percentage of patients with biochemical response was higher in the seladelpar versus placebo group.
An asymptomatic 55-year-old woman presented with thrombocytosis discovered 2 years earlier. Laboratory testing revealed the following: hemoglobin 13.1 g/dL, red blood cell count 5.04 ×…
Casgevy is the first CRISPR-based medicine to be approved for use in the United States for sickle cell disease and beta-thalassemia.
Cheryl Schwartz discusses Takeda’s report that calls for enhanced health equity and timely diagnosis for rare disease patients
Interdisciplinary research team using AI, engineering, and knowledge of disease complications to create “CART” STONY BROOK, NY, April 25, 2024 – The debilitating disease of…
The former N.F.L. player has been living with A.L.S. for more than a decade. Sharing “the most lacerating and vulnerable times” in “A Life Impossible”…