TreatSMA and charity meetings
TreatSMA had a meeting today with SMA Support UK and The SMA Trust due to the current meetings being held by EMA and their decision expected to be made very soon.
Biogen updates SMA Support UK and SMA Trust
Biogen have provided the following update to the SMA Support UK and SMA Trust in response to their queries:
Biogen update on efficacy of nusinersen
In infants with spinal muscular atrophy (SMA), treatment with Spinraza (nusinersen), the first FDA-approved drug for SMA, reduces the risk of death or permanent ventilation, according to new results from the Phase 3 ENDEAR study (NCT02193074).
European procedure for registration of medicines
The evaluation of medicines in the EU can be through single national submissions (in the first instance then a mutual review lead by the country who made the first assessment) but in the case of medicines of high importance like…
Nusinersen clinical trial update: Boy with type 1 SMA progressing well
This video from Nemours Children’s Hospital shows the effects of the drug nusinersen on a young spinal muscular atrophy (SMA) patient.