MHRA Foreword

I had the great pleasure of meeting Chris Kessler recently and I am so grateful to him for his bravery and eloquence in sharing his son Charlie’s story. This is a story of breakthrough medical advances giving the hope of a happy and healthy life to children like Charlie, when in previous generations they would have had few if any successful treatment options. It highlights the crucial role that we can play as regulators to ensure that the latest treatment options for rare diseases are available to patients in this country and beyond. It is for those patients, and their families in the case of children such as Charlie, to choose what is right for them, but they can only have those choices available to them with good regulation that prioritises treatments for rare diseases. We

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