Advances in Sickle Cell Disease
Dr. Smith-Whitley, pediatric hematologist at the Children’s Hospital of Philadelphia, discusses the advances in sickle cell disease.
Dr. Smith-Whitley, pediatric hematologist at the Children’s Hospital of Philadelphia, discusses the advances in sickle cell disease.
The NFL says it will allow players to wear protective soft-shell helmet covers known as…
Ten years after dipping its toe into the gene therapy pool in a licensing deal with Spark Therapeutics, Pfizer has gained an FDA approval for…
The clinic at East Tennessee State University Health is the only one in the state to offer free multidisciplinary care to ALS patients.
There has been a breakthrough in the research on the disease amyotrophic lateral sclerosis (ALS). Scientists at Umeå University report that the disease progression in…
In-line sales for Alzheimer’s drug Leqembi and a beat for rare-disease drug Skyclarys has Wall Street giving Biogen another chance.
EverythingALS is a patient-focused non-profit, part of Peter Cohen Foundation (PCF) a 501(3)c organization. Our mission is to support efforts to care for ALS patients…
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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