Collaboration Between C-Path and NORD will Help Standardize Data and Accelerate Research for Rare Diseases

Critical Path Institute The Critical Path Institute, or C-Path is a nonprofit organization which was established in 2005. It has a US branch, headquartered in Tucson, Arizona as well as…

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Results From First Patient Treated in Epidermolysis Bullosa Phase 2 Trial are Extremely Promising

Epidermolysis Bullosa Epidermolysis bullosa (EB) is a rare condition affecting approximately 500,000 individuals globally. Patients in the United States and Europe account for 50,000 of these individuals. The hereditary condition is…

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The Drug Abatacept Appears Beneficial in Systemic Sclerosis Despite Missing Primary Endpoint
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The Drug Abatacept Appears Beneficial in Systemic Sclerosis Despite Missing Primary Endpoint

According to a story from Medpage Today, a recent study testing the medication abatacept (marketed as Orencia) as a treatment for diffuse cutaneous systemic sclerosis concluded with rather mixed results.…

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Researchers Might Have Just Made a Breakthrough Discovery for Treating Rare Proteinopathies

It's common knowledge among researchers who study diseases called toxic proteinopathies that this group of disorders are the result of misfolded proteins that reside in cells. Certain proteins fail to…

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Company Cleared to Begin Trials for Experimental Retinitis Pigmentosa Drug

According to a story from globenewswire.com, the drug developer ProQR Therapeutics N.V. has recently announced that the US Food and Drug Administration (FDA) has officially cleared the company's Investigational New…

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Attention Biotech and Stem Cell Researchers: Two Potential Grant Programs!

Patient Worthy wishes to share the following funding opportunities with faculty/ biotech having interest in these areas of research. The two grant programs were announced by the Orphan Disease Center…

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