Study Suggests Rituximab Effective for Refractory Juvenile Myasthenia Gravis
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Study Suggests Rituximab Effective for Refractory Juvenile Myasthenia Gravis

  In many cases, autoimmune disorders like myasthenia gravis are treatable. However, there are some cases of refractory (treatment resistant) myasthenia gravis that presents an unmet patient need. However, researchers…

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ICYMI: Good Results From Evinacumab for Rare Form of Familial Hypercholesterolemia
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ICYMI: Good Results From Evinacumab for Rare Form of Familial Hypercholesterolemia

  A press release issued recently by Regeneron Pharmaceuticals heralded the acceptance of a Biologics License Application by the FDA for Priority Review of its investigational drug, evinacumab. The Priority…

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European Commission Grants Orphan Designation Designation for Investigative Achondroplasia Therapy
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European Commission Grants Orphan Designation Designation for Investigative Achondroplasia Therapy

Ascendis Pharma has just announced that their investigative therapy for achondroplasia called TransCon CNP has just received Orphan Drug Designation from the European Commission. This same designation has already been…

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Testing the Safety and Efficacy of Tradipitant for Patients with Diabetic and Idiopathic Gastroparesis
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Testing the Safety and Efficacy of Tradipitant for Patients with Diabetic and Idiopathic Gastroparesis

by Danielle Bradshaw from In The Cloud Copy Gastroparesis is a condition in which your stomach’s muscles aren’t able to completely move food through your stomach. Symptoms of gastroparesis include…

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These Organizations Want to Give an Adaptive Bicycle to Every Disabled Child
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These Organizations Want to Give an Adaptive Bicycle to Every Disabled Child

  Zacari was born with a rare neuromuscular disorder called arthrogryposis multiplex congenita. Children with the disease are unable to straighten or bend their legs or arms. Yet, according to…

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