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 […]
Genetics
In a Heartbeat: Understanding autoinflammation (Pittsburgh)
Children’s Hospital of Pittsburgh of UPMC has recruited Dr. Scott Canna to research autoinflammatory disorders, which can cause the body’s immune system to attack its […]
Researchers Unlock An Immunity ‘Black Box’
A research team led by St. Jude Children’s Research Hospital immunologists has revealed a previously unknown immune machinery that goes awry to trigger the inflammatory […]
Ultra-rare disease drugs: has access in England just got harder?
Story from Andrew McConaghie on PharmaPhorum New rules on market access introduced by NICE from 1 April will affect most new medicines – but gaining […]