Two of the three available seats for patient experts taken by TreatSMA trustees for the Risdiplam appraisal taking place on the 11th May 2021.

A new clinical trial is starting for gene therapy for patients who are: Symptomatic SMA diagnosis based on gene mutation analysis with bi-allelic survival motor neuron 1 (SMN1) mutations (deletion or point mutations) and any copy of the survival motor neuron 2 (SMN2) gene. Weight ≥ 8.5 kg and ≤ 21 kg at the time…

A new clinical trial is starting for gene therapy for patients who are: Symptomatic SMA diagnosis based on gene mutation analysis with bi-allelic survival motor neuron 1 (SMN1) mutations (deletion or point mutations) and any copy of the survival motor neuron 2 (SMN2) gene. Weight ≥ 8.5 kg and ≤ 21 kg at the time…

TreatSMA’s short film competition launches today, get your entries in by the 14th April to be in with a chance of winning a £50 Amazon voucher!

TreatSMA’s short film competition launches today, get your entries in by the 14th April to be in with a chance of winning a £50 Amazon voucher!

With the news regarding EMA Approval we are aware there may be some questions/concerns regarding EAMS. Please see below the following statement we have received from Roche.

European Medical Agency has recommended that Risdiplam can be used for treatment of SMA. The approval scope: 5q SMA over 2 months with clinical symptoms of SMA type 1, 2 or 3 OR with 1–4 copies of the SMN2 gene.

With the news regarding EMA Approval we are aware there may be some questions/concerns regarding EAMS. Please see below the following statement we have received from Roche.