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Exondys 51 is not a cure, but it has the potential to slow down the progression of this devastating condition, which would buy families valuable time and keep children walking for longer.
Catherine Woodhead, Chief Executive Officer at Muscular Dystrophy UK, said:
Exondys 51 is not a cure, but it has the potential to slow down the progression of this devastating condition, which would buy families valuable time and keep children walking for longer. We know this decision will be a blow to families and we share their disappointment.
It is encouraging to hear that Sarepta remains committed to bringing this treatment to Europe, and knowing there is a drug out there with the potential to help children walk for longer gives hope for a better future. It is an encouraging time for research and we know there are treatments on the horizon. We will keep pushing to ensure people can access these quickly.