New target identified for potential ALS therapy

Amyotrophic lateral sclerosis (ALS) is a devastating neurodegenerative disease that affects motor neurons.

Researchers find new genes that contribute to hypoplastic left heart syndrome

By identifying genes in patients and testing their effects in fruit flies, researchers from Sanford Burnham Prebys have found new genes that contribute to hypoplastic

July 18, 2023

Potentially Protective Agent Shows Benefit in Niemann-Pick Type C

Patients with Niemann-Pick disease type C who received N-acetyl-L-leucine (NALL), an agent that potentially ameliorates lysosomal and metabolic dysfunction, showed reduced neurologic signs and symptoms

February 1, 2024

NanoSyrinx appoints Anthony Johnson as Non-Executive Director

Appointment of Big Pharma executive strengthens leadership team to drive ambitious growth strategy.

November 13, 2023

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