According to a story from the National Gaucher Foundation, the stress and difficulties of living with a rare or chronic disease can take a major toll on a patient's mental…
According to a story from CNBC, the now former Commissioner of the US Food and Drug Administration (FDA) Scott Gottlieb was recently featured on the news network for a discussion…
According to a story from Parkinson's News Today, a study evaluating a pool of phase 3 clinical data confirmed the effectiveness of Gocovri, a drug that was approved by the…
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 Acrofan, the biotechnology company Moderna, Inc. recently released the results of a preclinical study which described the use of mRNA which encodes a monoclonal antibody…
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…
According to a story from Financial Buzz, the drug developer Rafael Pharmaceuticals, Inc., recently announced that a Phase II clinical trial that will test the company's experimental drug CPI-613 in…
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The Asco Post recently interviewed Dr. Jean Pierre Bizzari, the Executive Vice President of Celgene, a multinational bioparmaceutical company. The interview covered various aspects of research and development (R&D)…
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 Motherhood In-Style Magazine, Miley Timmermanns was only two years old when she was first diagnosed with an extremely rare disorder: Cornelia de Lange syndrome. Parents…
According to a story from Central Charts, the drug developer Crinetics Pharmaceuticals, Inc. recently issued an announcement revealing that the company has begun the initialization of a Phase 1 clinical…
When you or your child is first diagnosed with a rare disease it can be so difficult to know where to turn. You want to learn everything about the condition,…
According to a story from globenewswire.com, the biopharmaceutical company Abeona Therapeutics, Inc. recently issued an announcement in regards to the company's Phase 1/2 clinical trial, which is testing the company's…
According to a press release from the British biotechnology company GW Pharmaceuticals, the company has reported successful results at the conclusion of its phase 3 trial of tuberous sclerosis complex…
My name is Antonio Maltese and I am a 22 year old senior Political Science major concentrating in International Relations and minoring in German at Virginia Commonwealth University. Back in…
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…
According to a story from Healio, a recent study suggests that the addition of four more genes to the conventional genetic test used to diagnosed familial hypercholesterolemia could allow more…
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 MD Magazine, a recent study that was presented at Digestive Disease Week 2019 suggests that patients with ulcerative colitis, a rare form of inflammatory bowel…
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…
Q&A with NORD Director of Membership, Debbie Drell, on the Living Rare, Living Stronger Patient and Family Forum, June 21-23 in Houston, Texas Which health care professionals would most benefit…
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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 Daily Bruin, a team of scientists from UCLA are in the midst of developing the first therapy for multiple sclerosis that could address the…
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