Translarna can treat Duchenne caused by a nonsense mutation (about 10-13% of cases). The drug is available in England and Wales via a Managed Access Agreement – which gives patients access to the drug, while more data is collected about its effectiveness.
The latest update on the Translarna Managed Access Agreement focuses on the COVID-19 outbreak. It says that wherever it is safe and appropriate people should continue to receive Translarna as normal.
Translarna will still be delivered by homecare companies, but these companies are much busier than normal. This means your delivery schedule might change – if you’ve got questions the homecare provider will be able to answer them.
The data collection required by the MAA may not be possible during the COVID-19 outbreak. The NHS has put in place plans to use video and telephone technology to monitor those receiving the drug. As part of this, you may be asked to perform a physiotherapy assessment and other simple tests via video. If this isn’t possible, the tests will be done as soon as possible in the clinic; the usual rules about frequency of clinic visits will be relaxed if necessary.
For those who are eligible to start taking Translarna, it is possible that there may be some delays in receiving treatment depending on the availability of clinical staff. To enter the MAA, an initial set of data must be collected and the rules about this data collection have been relaxed to ensure that as many people as possible have access to the drug as quickly as possible.
You can read the full statement here.
We also have a detailed FAQ about Translarna and the MAA
Physiotherapy webinar 2 with Marion Main (making stretches fun)
