Next-generation metagenomic sequencing test developed at UCSF proves its effectiveness in quickly diagnosing almost any kind ...
CPCT provides free lifelong access to Risdiplam for 50 children with SMA, supported by Roche Pharma India and Karnataka government.
As of last year, Clementine was just one of 10 people who have been diagnosed with a mutation to the TLR8 gene. She received ...
Are you battling a rare disease or think you might be suffering from one? If so, there are resources to get help from ...
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Repurposed FDA-approved drug could uncover potential treatment for rare genetic disordersTay-Sachs disease, the more common of the two disorders, typically manifests within the first year of life, progressing ...
A video going viral on TikTok is raising questions about a North Carolina lawmaker’s alleged response to a constituent.
In a groundbreaking study, researchers at McMaster University have identified a potential treatment for Sandhoff and Tay-Sachs diseases—two rare, often fatal lysosomal storage disorders that cause ...
For some, an even bigger draw than the silver, furniture and artwork at Michael Corcoran’s auctions is the 96-year-old ...
Healx will use its AI-based drug discovery tech to analyze proprietary Sanofi compound data and identify potential rare disease targets.
Less than 1% of infants are born with short bowel syndrome, which happens when either the large or small intestine doesn’t ...
The combined net Q3 2024 revenue of approximately $3.1 million for prescription and non-prescription products, including license revenue, increased approximately 14% versus net Q2 2024 revenue of $2.7 ...
California’s recent decision to create a Rare Disease Advisory Council is a milestone that brings new hope to people living ...
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