According to a release from Sangamo Therapeutics and published by PR Newswire, experimental severe hemophilia A drug SB-525 proved to be well-tolerated and generally safe in a phase 1/2 clinical…
GT Biopharma has just announced that their Phase 1 clinical trial for Mastocytosis, Myelodysplastic Syndrome (MDS), and Acute Myeloid Leukemia (AML) has been authorized to begin. Although this investigation had previously…
Happy April! We hope everyone's month is going well! This week, we're highlighting two patient stories and two research stories. First, we have the story of Carolyn, a pemphigus patient…
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A recent article in the publication CheckOrphan announced encouraging results of a long term extension study sponsored by Ultragenyx involving UX007 (triheptanoin) in patients with long-chain fatty acid oxidation disorders…
Fabry Disease Fabry Disease is a rare lysosomal storage condition. Mutations in the GLA gene cause a type of fat called globotriaosylceramide to build up in the body's cells. Symptoms…
According to a publication from Medical News Today, in animal studies, a failed cancer drug showed signs of promise in treating frontotemporal dementia. Frontotemporal Dementia Dementia is a blanket term…
According to a story from Healio, there are a number of recent reports of progress in the development of treatments for rare forms of liver disease. Many rare liver diseases…
Bruce Alan Fries, President of the Patient Centered Care Advocacy Group, along with Holly Ahern and Chris Fisk from the Lyme Action Network, have issued an official "Information Quality Request…
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