Living with a rare disease: a husband and father’s perspective
What does rare disease mean to you? To me, rare disease means a mutation called R1016X in a gene called NLRP12 on my son’s and […]
ultra-rare
A New Gene Therapy Cure Just Treated Its First-Ever Patient
Rarefied Cures This March, a child with severe combined immune deficiency (SCID) became the second commercial gene therapy patient ever. The patient started receiving a drug […]