This Just in: Data from Phase 3 Trial for Familial Adenomatous Polyposis is Coming Early 2019
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This Just in: Data from Phase 3 Trial for Familial Adenomatous Polyposis is Coming Early 2019

Familial adenomatous polyposis (FAP) is a condition which causes extra tissue to form in various parts of the body such as the large intestine and gastrointestinal track. FAP affects about…

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Device Allows Monitoring of Lung Function at Home For Duchenne Muscular Dystrophy Patients
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Device Allows Monitoring of Lung Function at Home For Duchenne Muscular Dystrophy Patients

According to a story from EurekAlert!, monitoring lung function is an important role of managing Duchenne muscular dystrophy and other diseases that can cause wasting of the respiratory muscles. Many…

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How Planning a Wedding Solved the Riddle of a Sister’s Mysterious Illness
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How Planning a Wedding Solved the Riddle of a Sister’s Mysterious Illness

The Heritage Florida Jewish News reported a story about one sister's journey to her Gaucher Disease diagnosis, thanks to her older sister's wedding prep. What is Gaucher Disease? Gaucher disease…

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New Open Data Portal Will Improve Neurofibromatosis Research
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New Open Data Portal Will Improve Neurofibromatosis Research

According to a story from EurekAlert!, a collaboration of Sage Bionetworks, The Children's Tumor Foundation (CTF), and the Neurofibromatosis Therapeutic Acceleration Program (NTAP) have announced the initiation of the very…

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Possible Treatment for Spinal Muscular Atrophy Gains PRIME Designation From the EMA
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Possible Treatment for Spinal Muscular Atrophy Gains PRIME Designation From the EMA

According to a story from pm360online.com, the pharmaceutical company Roche recently announced that its investigational therapy risdiplam has awarded PRIME designation from the European Medicines Agency (EMA). The drug has…

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After Being Ignored by Doctors, Former NFL Cheerleader Accurately Diagnoses Own Pectus Excavatum
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After Being Ignored by Doctors, Former NFL Cheerleader Accurately Diagnoses Own Pectus Excavatum

According to a story from NTD, a former NFL cheerleader successfully diagnosed her own rare disease, pectus excavatum, after her symptoms were dismissed by her doctors as nothing more than…

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Markelle Fultz of the Philadelphia Sixers was Diagnosed With Thoracic Outlet Syndrome. What Does This Mean?
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Markelle Fultz of the Philadelphia Sixers was Diagnosed With Thoracic Outlet Syndrome. What Does This Mean?

According to a story from philly.com, Markelle Fultz, who plays as a guard for the Philadelphia 76ers, was recently diagnosed with thoracic outlet syndrome. It has not been good news…

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Phase 1 Gene Transfer Trial for Recessive Dystrophic Epidermolysis Bullosa is Recruiting Patients
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Phase 1 Gene Transfer Trial for Recessive Dystrophic Epidermolysis Bullosa is Recruiting Patients

According to a story from Debra of America, a new Phase 1 clinical trial being operated by Stanford University is currently recruiting participants. This trial will be a test of…

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