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 ...
A new study reveals a critical vulnerability in a brain receptor targeted by a rare autoimmune disease. Researchers have ...
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 ...
Lozano is a rare disease mom, neuroscience Ph.D. candidate at UC Davis, and board member for the PURA Syndrome Foundation. In May, a historic moment in science and medicine was captured in a single ...
A “fit and healthy” woman from Surrey, UK, was left paralysed after years of headaches dismissed as stress were found to be ...
A "gene silencer" (technically known as small interfering RNA, or siRNA), locally delivered by nanoparticles embedded in an ...