Potential Gene Therapy for Late Infantile Batten Disease Earns Rare Pediatric Disease Designation

According to a story from BioSpace, the biotechnology company REGENXBIO, Inc. recently announced that its experimental gene therapy candidate called RGX-181 has been granted Rare Pediatric Disease designation from the…

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This Just In: Patient Reported Experiences are Important for Research in Osteogenesis Imperfecta

What is osteogenesis imperfecta? Osteogenesis imperfecta (OI) actually refers to a group of four distinct rare disorders- OI Type I, II, III, and IV. They're all characterized by brittle bones which…

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This Website Hopes to Help Women With Inflammatory Bowel Disease Have Healthy Pregnancies
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This Website Hopes to Help Women With Inflammatory Bowel Disease Have Healthy Pregnancies

According to a story from Reuters, a new website developed by the American Gastroenterological Association is meant to help women that have inflammatory bowel disease have safe and healthy pregnancies.…

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Why is it Difficult to Diagnose Chronic Inflammatory Demyelinating Polyneuropathy (CIDP)?
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Why is it Difficult to Diagnose Chronic Inflammatory Demyelinating Polyneuropathy (CIDP)?

According to a story from WebMD, the rare nervous system disorder chronic inflammatory demyelinating polyneuropathy is notorious for being difficult to diagnose. Many rare diseases can be challenging to identify.…

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Study Shows Treatment may be Effective for Chronic Kidney Disease but Increase Risk of Hyperkalemia

A recent study, called TOPCAT, evaluated the effects of spironolactone as a treatment for individuals living with Chronic Kidney Disease (CKD) who are also diagnosed with heart failure with preserved ejection fraction,…

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