RG7916 increases SMN protein 2.5-fold
PTC Therapeutics informed that the experimental drug RG7916 increased the SMN protein 2.5-fold in SMA type 2 and 3 patients who took part in the SUNFISH clinical trial.
Category: Updates
RG7916 increases SMN protein 2.5-fold
PTC Therapeutics informed that the experimental drug RG7916 increased the SMN protein 2.5-fold in SMA type 2 and 3 patients who took part in the SUNFISH clinical trial.
Unsatisfactory response from NHS England
TreatSMA has received a formal response from NHS England to the letter sent by the UK medical community about the Expanded Access Programme of nusinersen.
Unsatisfactory response from NHS England
TreatSMA has received a formal response from NHS England to the letter sent by the UK medical community about the Expanded Access Programme of nusinersen.
SMA Awareness Day
Today 30/9 the world celebrates SMA Awareness Day. Do you know what SMA stands for?
A new trial of gene therapy in SMA announced
Today Avexis announced the start of a new clinical trial of gene therapy in SMA.
SMA Awareness Day
Today 30/9 the world celebrates SMA Awareness Day. Do you know what SMA stands for?
A new trial of gene therapy in SMA announced
Today Avexis announced the start of a new clinical trial of gene therapy in SMA.