As for the newly published strategic collaborative approach, the EMA and FDA discuss possible ways to enhance the efficiency of medicine development in Gaucher disease, a rare lysosomal storage disorder.
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The Haystack Project is a 501(c)3 nonprofit that brings together patients organizations representing patients suffering from or caring for patients with extremely rare diseases. Our mission is to educate policymakers and other stakeholders about the need for policies that recognize the unique circumstances of extremely rare conditions and treatments and expand incentives critical to ensuring they can reach patients.
