Myositis Research Could Aid in COVID-19 Vaccine Development

Myositis Myositis is an autoimmune disease that leads to muscle damage. Patients live with chronic disability due to the symptoms of this rare condition. These include skin rashes, pain, weakness, fatigue,…

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New Drug Application for Niemann-Pick Disease Type C Treatment Submitted Under Rolling Review
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New Drug Application for Niemann-Pick Disease Type C Treatment Submitted Under Rolling Review

According to a story from globenewswire.com, the biopharmaceutical company Orphazyme A/S has recently announced that it has submitted its New Drug Application (NDA) for its experimental treatment arimoclomol to the…

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Arimoclomol Receives the Fast Track Designation for the Treatment of ALS
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Arimoclomol Receives the Fast Track Designation for the Treatment of ALS

CytRx Corporation has recently received the Fast Track designation for their ALS treatment, arimoclomol. This status was also given for the treatment of Niemann-Pick disease and sporadic Inclusion Body Myositis…

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Results from Phase II/III Trial Bring Hope for FDA Approval for New Niemann-Pick Disease Type C Treatment

Niemann-Pick Disease is a genetic lysosomal storage disease. Lipids accumulate in the lysosomes of cells, causing debilitating symptoms. There are three forms of the disease- A, B, and C. Each…

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This Company Just Endorsed New Patient Advocacy Guidelines
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This Company Just Endorsed New Patient Advocacy Guidelines

According to a story from globenewswire.com, the biopharmaceutical company Orphazyme has officially endorsed a set of guidelines that outline how rare disease patient advocacy organizations and drug companies should interact…

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Phase II Trial for Investigational Gaucher Disease Drug Begins
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Phase II Trial for Investigational Gaucher Disease Drug Begins

According to a story from GlobeNewsWire, the biopharmaceutical company Orphazyme A/S, which is committed to the development of treatments for patients that are living with rare diseases, has recently announced…

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