In honor of our annual AHC Day, AHCF is excited to announce that we will be able to fund two research projects to get us one step closer to a treatment for AHC!
Tomorrow, January 18th, is AHC day. AHC or alternating hemiplegia of childhood, is a devastating neurological disease caused by a mutation in the gene ATP1A3.
The study estimated that each year, 119,000 babies are born around the world with fetal alcohol syndrome.
Levi is 3-years-old and has LCMD, rare form of congenital muscular dystrophy (CMD).
Syringomyelia is a disease that is identified when a syrinx also termed cyst forms inside the spinal cord.
For those with rare diseases, it’s particularly critical to protect oneself against the viruses that can wreak havoc on the respiratory, digestive, and immune systems.
With Biogen's Spinraza for the treatment of spinal muscular atrophy being approved in late December, the data of the drug's phase 3 clinical trial was finally presented at an annual neurology meeting.
Bridge the Gap – SYNGAP Education and Research Foundation has launched the largest-ever study to research SYNGAP1.
Christine Ha has neuromyelitis optica (NMO), a disease that has left her blind since 2007. Christine is also a Master Chef winner, TV host, author, and in inspiration to many in the rare disease community.
BioMarin has transferred their Duchenne natural history database to the non-profit group, CureDuchenne.