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.
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.
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.
Calling all parents/carers of children under 12, children between the ages of 12-18 and also adults or carers of adults to participate in an ongoing study funded by Biogen.
Last week Novartis informed the patient community that earlier reports were incorrect and that the development of branaplam as a treatment for SMA will continue.
We are aware of today’s media reports saying that Novartis intends to halt the development of branaplam, a promising oral drug for SMA. We are unable to confirm these news at this stage. We expect to have more information in the following days.
During an election period, Government Departments are restricted in the announcements that they are able to make or the information that they are able to publish. The ongoing communication between NHS England and the Patient Advocacy Groups (PAGs) about implementation…