Discover Sanfilippo Syndrome a rare genetic disorder affecting childrens development Learn about its symptoms treatment ...

The daughter of a Kent County sheriff’s deputy who is fighting a rare genetic disorder will benefit from this year’s Robert ...

Tierney LaSotta was diagnosed with Sanfilippo Syndrome, a rare condition also known as Childhood Dementia because of its ...

Ultragenyx Pharmaceutical is a mid-cap biotech firm with a promising pipeline and an optimistic outlook from analysts. Click ...

JCR Pharmaceuticals Co., Ltd. (TSE 4552; "JCR") announced today that it will present at the 21st Annual WORLDSymposiumtm 2025, held February 3-7, 2025, in San Diego, Calif. The poster presentations ...

Denali’s candidate is its DNL12 enzyme replacement therapy (ERT) for mucopolysaccharidosis (MPS) IIIA, a form of lysosomal storage disease also known as Sanfilippo syndrome type A, which is in a ...

June 10, 2024 — Scientists have grown 'mini-guts' in the lab to help understand Crohn's disease, showing that 'switches' that modify DNA in gut cells play an ... Major Cause of Inflammatory ...

Optimizing Drug Packaging: Strategic Approaches for De-Risking Drug Development ...

Morning Coffee May Protect the Heart Better Than All-Day Coffee Drinking Jan. 7, 2025 — People who drink coffee in the morning have a lower risk of dying from cardiovascular disease and a lower ...