FDA approves 1st gene therapy for Sanfilippo Syndrome type A
Digest more
New framework brings together 108 rare lysosomal disorders, substantially expanding the number included in traditional ...
Ultragenyx said its experimental therapy for Angelman syndrome, a rare disease, showed no benefit compared to a sham treatment in a Phase 3 trial.
Researchers from Queen Mary University of London, the Berlin Institute of Health at Charité (BIH) and Genomics England have ...
Source: Mark L. Ruffalo, D.Psa., L.C.S.W. Psychosis has historically been defined as "the loss of contact with reality" and can be observed in various mental disorders, including schizophrenia, ...
Ayik is an advocate for people living with rare diseases. Recently, the smoldering embers of conflict in my family’s rare disease community have burst into flames over the use of an eponym: ...
This story discusses suicide. If you or someone you know is having thoughts of suicide, please contact the Suicide & Crisis Lifeline at 988 or 1-800-273-TALK (8255). A little-known neurological ...
Some results have been hidden because they may be inaccessible to you
Show inaccessible results