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 […]
Rare Disease
A Nuanced Message: Marketing to the Rare Diseases Community
Check traditional marketing strategies at the door, because when it comes to rare diseases they simply don’t apply. Experts say established methods of directing campaigns […]
PatientsLikeMe and Shire to co-develop rare disease patient network
PatientsLikeMe and Shire are to work together to better understand rare disease patient health and disease progression. PatientsLikeMe is now a well-established online community of […]
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 […]
NLRP12.com’s 2017 Scottish Council Manifesto released
For the 2017 Scottish Local Council Elections NLRP12.com have released their manifesto for council candidates throughout Scotland. We had hoped to be able to get […]
ICMR launches registry of rare diseases
Launched on April 27 by the Indian Council of Medical Research (ICMR), the registry is important for determining the precise number of patients, age of […]
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 […]