In the view of recent NHS plan we once again contacted NHS, Government and NICE highlighting that no significant changes in the policy has been made to accommodate rare diseases. The letter shows our frustration with the system and was…
This is important! If we are to get Spinraza, we must work hard to get it. Please complete this survey.
Today, the Polish minister for health announced that Spinraza will be available through the national health care in Poland.
Novartis announces FDA filing acceptance and Priority Review of AVXS-101, a one-time treatment designed to address the genetic root cause of SMA Type 1
Today TreatSMA along with SMA Support UK and Muscular Dystrophy UK sent another letter to NICE, in which we asked that nusinersen be placed on the agenda of the nearest Committee C meeting which will take place on 6 February.
SRK-015, an experimental drug for SMA being developed by the US biotechnology company Scholar Rock, has been awarded the Orphan Medicinal Product designation in the European Union.