Acid Sphingomyelinase Deficiency (ASMD) is a rare disease that limits and shortens the lives of children and adults. Breakthrough treatments can halt symptoms and reverse disease progression. Despite clear clinical benefits, many UK patients still cannot access these medicines, even though they are available in over 50 countries including France, Germany and Australia. Changing the way treatments like these are valued is the first step in giving UK patients with rare diseases a chance of an impr
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