TreatSMA sponsor Rare Disease Day event – 26 February 2020
TreatSMA are proud to announce that we will be one of the sponsors for Royal Holloway’s 10th Annual Rare Disease Day Event on Wednesday 26th of February.
Roche’s risdiplam showed significant improvement in motor function in people aged 2-25 with type 2 or 3 spinal muscular atrophy
Roche released part 2 of the SUNFISH data which shows significant improvement both type 2 and type 3 patients. Read on for further details.
Physio with Marion Episode 4
TreatSMA hosted another Physio with Marion session on Sunday, and thanks to Marion and all of those who took part in asking questions, it was another great success which saw questions and views from around the globe.
Risdiplam is effective in treating babies with SMA type 1
Risdiplam has been proven safe and effective in treating babies with SMA 1
Pre-approval access programme to risdiplam in the UK
Update on Roche's pre-approval access programme to risdiplam and how it will be implemented in the UK
Roche makes risdiplam available globally through a pre-approval access programme
Today Roche announced that it is making risdiplam available through a global Pre-Approval Access/Compassionate Use Programme. Under the programme, the company will provide risdiplam for patients who cannot receive an approved treatment.
Physio with Marion Episode 3
TreatSMA hosted another Physio with Marion session on Sunday, and thanks to Marion and all of those who took part in asking questions, it was another great success which saw questions and views from around the globe, over 1000 by the end of the evening.
Zolgensma to be made available globally under a managed-access programme
Avexis has informed us that it will shortly make their gene therapy drug Zolgensma available for free to a limited number of eligible patients outside of the United States. A new managed-access programme of Zolgensma will be opened globally but it is uncertain whether UK will be included.