Risdiplam Delivers An Improvement to Infants with Spinal Muscular Atrophy Type 1
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Risdiplam Delivers An Improvement to Infants with Spinal Muscular Atrophy Type 1

Genentech (Roche Group) announced positive results of its FIREFISH trial in a recent Business Wire news release.  The second part of the study evaluated risdiplam’s efficacy on infants ages one…

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Blood Thinners Improves Quality of Life With Idiopathic Pulmonary Arterial Hypertension, Worsens Those with Other Variation

A medication that works for some patients with a rare disease may not be effective for others, even with the same disease. For rare diseases, this is important because sample…

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Acromegaly Community to Host Patient-Focused Drug Development Meeting with FDA
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Acromegaly Community to Host Patient-Focused Drug Development Meeting with FDA

On April 28th, 2020, Acromegaly Community, a nonprofit organization that is dedicated to supporting acromegaly patients, will be hosting a patient focused drug development meeting (PFDD). These meetings are an…

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