Searching for a cure, Improving lives
Data published today in the journal Molecular Therapy demonstrates initial clinical proof of principle gene therapy to increase muscle strength and function in Becker muscular dystrophy patients. All six of the patients were followed at least six months and three of the patients were followed for one year with no drug-related adverse events reported. On
This afternoon witnesses the launch of Genetic Alliance UK’s second Patient Charter: “Patient perspectives and priorities on NHS England’s commissioning of medicines for rare diseases”. After participating as an integral member of work groups leading to the Charter’s formation, Action Duchenne will be attending the launch at a sell-out event at Great Ormond Street Hospital. This
This week “Disabled People Against Cuts” held a vigil outside outside the Law Courts in support of a second court case against the Department..
After Tory Peer Lord Freud last week mused over whether people with disabilities should work for two pounds an hour, guardian journalist Aditya..