In the United States, Fast Track designation is granted to drugs designed to treat rare and/or serious conditions and that fill an unmet medical need. The process aims to…
Dr. Valeria Ricotti of the Institute for Child Health- Great Ormond Street UK, spoke with BBC News on the subject of AI analyzing body movement. Dr. Ricotti and other researchers…
The number of identified rare diseases continues to climb every year, with the number recently reaching the 11,000+ mark. Some of them have only a handful of identified cases, and…
For many people living with mantle cell lymphoma (MCL), the standards-of-care include Bruton's tyrosine kinase (BTK) inhibitors. But there is a subset of patients who do not respond well,…
Enzyme replacement therapy (ERT) has been explored as a potential therapeutic option for mucopolysaccharidosis type III A (MPS III-A or Sanfilippo syndrome type A). However, these solutions have not…
In a late December 2022 news release from biopharmaceutical company Altimmune, Inc., the company shared that positive topline data was available from a 24-week study—including a 12-week extension—which evaluated…
From November 10-14, 2022, various stakeholders within the rheumatology sphere connected at the American College of Rheumatology’s (ACR) Convergence 2022. During ACR Convergence, described as the world’s premier rheumatology experience,…
Currently, the standards-of-care for lupus include anti-malarial drugs such as hydroxychloroquine, corticosteroids and immune-suppressive therapies, and biologics such as Benlysta. But new therapies are urgently needed, especially as anywhere between…
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