Huntington's Disease Huntington's disease (HD) is a rare neurodegenerative disease. It progressively affects the patient's mobility and cognitive function. Unfortunately, a cure has yet to be discovered and the condition…
According to a story from PR Newswire, the biopharmaceutical company Respivant Sciences has recently announced that it has dosed the first patient in its phase 2b clinical trial. This trial…
According to a story from globenewswire.com, the specialty pharmaceutical company Santhera Pharmaceuticals will be entering a collaborative partnership with the University of Basel's Biozentrum. The goal of this partnership will…
The International Waldenstrom's Macroglobulinemia Foundation (IWMF) is a nonprofit organization founded by patients. Their mission? Support patients currently living with Waldenstrom's Macroglobulinemia (WM) while simultaneously supporting the search for a…
Happy Thursday! We hope everyone's enjoying the spring weather. Today, we're highlighting a story from a young man advocating for Huntington's patients. Next, we have an article about a surprising…
According to a story from thejournal.ie, Ireland's Health Services Executive (HSE) is facing renewed pressure from activists and patient advocates for a reasonable coverage decision regarding Spinraza, which is currently…
Orencia Orencia (abatacept) is a treatment for juvenile idiopathic arthritis (JIA) that was approved for use in the U.S. for the pediatric patient population in 2008. In Japan, the therapy…
According to a story from BioPortfolio, dogs have been making the lives of human beings better for thousands of years in many different ways, and now there is new one…
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Google’s Parent Company (Alphabet) Funds New Gene Editing Company An article recently published in MIT Review reports that Verve Therapeutics announced that it has received funding from Alphabet’s GV venture fund plus…
According to a story from The Washington Post, a host of different factors ultimately led to the tragic death of Olivia Shea Paregol, a freshman at the University of Maryland.…
According to a publication from Express Digest, Britain's National Health Service (NHS) has finally reached an agreement with American biotechnology company Biogen Inc. over the price of Spinraza - a…
According to a story from globenewswire.com, the biopharmaceutical company Poseida Therapeutics, Inc. recently announced that the company's investigational product candidate P-BCMA-101 has been granted Orphan Drug Designation from the US…
Short Bowel Syndrome Short bowel syndrome (SBS) is a rare condition which occurs when patients have a large portion of their intestine removed. The surgery which leads to this diagnosis…
According to a story from romania-insider.com, there are a total of 26 known cases of hereditary transthyretin mediated (HTTR) amyloidosis known in the country of Romania. However, 20 of these…
The Word "Rare" Unfortunately, many experts in rare disease believe the phrase "rare disease" may be harming the very population it describes. The Director of the National Center for Advancing…
Coronado is a resort city located in San Diego, California. It's known for its luxury hotels, delicious restaurants, beautiful parks, and gorgeous beaches. It has just been announced that the…
SCID Severe combined immunodeficiency (SCID) is a rare disease which causes patients to have little to no immune response. With practically no immune system, SCID patients are very susceptible to…
The Origins of Fighting H.A.R.D. The Fighting H.A.R.D. Foundation was established in 2015 by two sisters who both live with multiple rare diagnoses. 16-year-old Allie is diagnosed with juvenile rheumatoid…
According to a story from BioSpace, the drug developer Biogen recently released interim results from a Phase 1/2 clinical trial. This clinical trial is testing the drug tofersen, which is…
This week, a press release from the office of South Carolina senator Lindsey Graham announced the designation of May 17th as Glioblastoma Awareness Day. The Senate Resolution was cosponsored by…
PREFER PREFER stands for Patient Preferences in Benefit-Risk Assessments during the Drug Life Cycle. It is a multi-stage project developed by Newcastle University's Welcome Centre for Mitochondrial Research in effort…
Phage Therapy Phages, or bacteriophages, are the natural enemy of viruses. They are the most abundant organisms in the biosphere. Researchers have used phages as treatments before, however after the…
According to a story from globenewswire.com, the biopharmaceutical company Poseida Therapeutics, Inc. recently announced that the company's investigational product candidate P-BCMA-101 has been granted Orphan Drug Designation from the US…
Happy Wednesday! We're officially halfway through May if you can believe it! This week we're highlighting a piece about an intern using their rare experience to inform their work, followed…
The FDA has just recently approved CYRAMZA as a treatment for those with hepatocellular carcinoma (HCC), who have been treated with sorafenib, and who have ≥400 ng/mL of alpha-fetoprotein (AFP).…
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