This Company is Getting Behind Two New Von Willebrand Disease Clinical Trials
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This Company is Getting Behind Two New Von Willebrand Disease Clinical Trials

According to a story from BioPortfolio, the drug company Octapharma USA sponsored the 71st Bleeding Disorder Conference, which is put on by the National Hemophilia Foundation. The company is also…

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Phase 3 Clinical Trial for Amyotrophic Lateral Sclerosis Has Completed Enrollment
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Phase 3 Clinical Trial for Amyotrophic Lateral Sclerosis Has Completed Enrollment

BrainStorm Cell Therapeutics has just announced that they have completed enrollment in their Phase 3 clinical trial for amyotrophic lateral sclerosis (ALS). Enrollment was initiated way back in October of…

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Progressive Fibrosing Interstitial Lung Disease Drug Candidate Ofev Granted Breakthrough Therapy Status
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Progressive Fibrosing Interstitial Lung Disease Drug Candidate Ofev Granted Breakthrough Therapy Status

According to a publication from Scleroderma News, the American Food and Drug Administration (FDA) recently granted Breakthrough Therapy status to Ofev (generic name nintedanib), as a treatment for progressive fibrosing…

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Data Indicates That Gene Therapy for Spinal Muscular Atrophy Could be Useful for Older Patients
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Data Indicates That Gene Therapy for Spinal Muscular Atrophy Could be Useful for Older Patients

According to a story from MedCity News, interim data from a phase 1/2 clinical trial testing Zolgensma, a gene therapy for spinal muscular atrophy, in patients between the ages of…

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Idiopathic Intracranial Hypertension: Symptoms are Common but the Disease is Rare
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Idiopathic Intracranial Hypertension: Symptoms are Common but the Disease is Rare

  Jessica Walton described the staff at Queen Elizabeth Hospital in King’s Lynn as being in “a bit of a panic” when they thought that she was having a stroke.…

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Two Nonprofits Award $200,000 Grant for Development of Duchenne Muscular Dystrophy Patient Reported Outcomes
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Two Nonprofits Award $200,000 Grant for Development of Duchenne Muscular Dystrophy Patient Reported Outcomes

According to a story from PR Newswire, the nonprofit groups Duchenne UK and Parent Project Muscular Dystrophy (PPMD) have jointly awarded grant funding to the tune of $200,000 to Dr.…

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Amgen is Utilizing Technology to Improve Diagnosis and Adherence in Rare Disease

Diagnosis, Treatment, and Adherence There are many factors necessary for an individual to be able to thrive with a serious medical diagnosis. First, they must receive the correct diagnosis so…

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