Rare Disease
A Boy with an Extremely Rare Disorder was Finally Diagnosed Thanks to a Typo
According to a story from the TGen Blog, a simple typing mistake lead to a diagnosis for a boy with an incredibly rare genetic disorder.
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This Company is Getting Behind Two New Von Willebrand Disease Clinical Trials
- October 18, 2019
Phase 3 Clinical Trial for Amyotrophic Lateral Sclerosis Has Completed Enrollment
AI Company Healx Raises $56 Million Towards Treating Rare Diseases
Progressive Fibrosing Interstitial Lung Disease Drug Candidate Ofev Granted Breakthrough Therapy Status
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Rare Disease
Editor’s Choice: Advocating for Equitable and Accessible Health Care
Happy Thursday! It’s cold and flu season around our parts, so we hope everyone reading this has dodged the fall viruses! This week, we’re sharing
Systemic Scleroderma
Phase 1 Study of Experimental Systemic Sclerosis Treatment Bermekimab Begins
According to a recent press release from XBiotech, a Texas-based biotechnology company, patient dosing is now underway in a phase 1 clinical study of the
Rare Disease
NIH Commits Millions in Grant Funding for Rare Disease Research
According to a story from news-medical.net, the National Institutes of Health (NIH) has doled a total of $31 million on grants this year to 20
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Wilson Disease
The Next Frontier for Artificial Intelligence: Diagnosing Disease and Designing Drugs
A recent article in Forbes highlights a study on diagnostic performance published in The Lancet Digital Health. The study represents the first review to compare
Psoriatic Arthritis
A Drug Used to Treat Psoriasis Could be Effective for Ulcerative Colitis
According to a story from Medical News Bulletin, a recent study that was first published in the New England Journal of Medicine appears to have revealed a
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We believe rare disease patients are people, not a diagnosis. Through education, awareness and some humor, we help patients, caregivers and support persons by providing relevant and often inspirational news and stories.
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