Huntington’s disease is a rare inherited neurological disorder that progressively affects movement, cognition and mental health due to a genetic mutation ...
Actor turned rare disease advocate Luke Rosen tells how his daughter, Susannah, lives with a KIF1A-associated neurological ...
A new study has brought scientists one step further in the direction of developing a cure for a brutal group of rare brain disorders known as SYNGAP1-related disorders, or SRDs. Researchers were able ...
Jonathan Hughes, diagnosed with rare Progressive Supranuclear Palsy, pursues his dream of completing a 5K race with community support in San Diego.
A new study reveals a critical vulnerability in a brain receptor targeted by a rare autoimmune disease. Researchers have ...
A “fit and healthy” woman from Surrey, UK, was left paralysed after years of headaches dismissed as stress were found to be ...
OREGON, USA — Nearly two decades after first being identified, a rare and life-threatening brain-inflammation disease may soon have new treatment candidates. The disorder, called anti-NMDA ...
A "gene silencer" (technically known as small interfering RNA, or siRNA), locally delivered by nanoparticles embedded in an ...