2020 Dem Presidential Candidates Quietly Debate Rare Disease Policy
Seems like we JUST had a presidential election, right? But we're in the dead of summer 2019, inching closer to a presidential election that will once again take over our…
Seems like we JUST had a presidential election, right? But we're in the dead of summer 2019, inching closer to a presidential election that will once again take over our…
Happy Fourth of July Season! Today, we're highlighting stories about research and social media, robots in caregiving, sisters carrying each others' babies, and AI advancing glioblastoma research. After this past…
Dicerna Pharmaceuticals has just announced that they have submitted a Clinical Trial Authorization application for their investigational therapy DCR-A1AT. This application has been submitted to the Swedish Medical Products Agency…
According to a story from BioPortfolio, the biopharmaceutical company X4 Pharmaceuticals, Inc. has recently initiated a phase 3 clinical trial that is testing its current lead product candidate mavorixafor. This…
According to a publication from the Warwick Beacon, friends and family of 4-year-old Banner Mazza are raising money for the boy's medical expenses. Mazza was recently diagnosed with ROHHAD, a…
According to a press release from the Texas Biomedical Research Institute (Texas Biomed), researchers from the Institute have identified another new genetic mutation that they believe can cause rare congenital…
According to a story from The Boston Globe, the drug developer Ovid Therapeutics plans to move forward with a late stage clinical trial that will test the company's investigational drug…
Inflammatory Bowel Diseases Inflammatory bowel diseases (IBD) is the overarching term for both ulcerative colitis and Crohn's disease. In Canada, there are around 270,000 individuals living with the one of…
According to a story from Pharmaceutical Technology, the drug developer FibroGen's phase 3 clinical trial testing pamrevlumab as a treatment for idiopathic pulmonary fibrosis could be in trouble. While the…
According to a story from Newswise, scientists affiliated with the Huntsman Cancer Institute (HCI) of the University of Utah have found in recent lab studies that the experimental drug selinexor…
According to a press release from the California Institute for Regenerative Medicine (CIRM), the Institute's governing body has approved a grant of nearly $12 million to University of California, San…
According to a story from Markets Insider, the biopharmaceutical company Camurus has recently issued an announcement indicating that the US Food and Drug Administration (FDA) has accepted the company's Investigational…
According to a story from The Columbian, twins Jill Noe and Whitney Bliesner had developed a deep bond during their childhood days growing up in Oregon. However, they did not…
Anita Lawson was paying the doctor a visit for her annual checkup in 2003 when she was told something that surprised her: she had anemia. She had some issues with…
Emily Dover This is Emily Dover's story. At birth- perfectly healthy newborn, smaller than her siblings (8 pounds) 1 week- was having trouble sleeping and began crying in pain 4…
In May 2016, I was diagnosed with a rare genetic neurological disease called adrenoleukodystrophy (ALD). Sadly, it's incurable and there's no medication to ease my condition. Since then, my life…
According to a story from EurekAlert! an international team of scientific researchers have made an unusual discovery. The team found in the course of their studies that dietary supplementation with…
China has been making waves lately, improving its efforts to efficiently and effectively provide healthcare for its nearly 1.5 billion population. “Health is a prerequisite for people's all-round development and…
Happy Thursday! Today, we're highlighting stories on three patients facing different struggles: a professor, a Jeopardy host, and a man from New Jersey. We also have an article about a…
Tannaz Motevalli Tannaz Motevalli is the Exhibition Coordinator at the National Library of Medicine, an artist, a researcher, and a writer. She also lives with cystic fibrosis (CF). Experiencing fewer…
In honor of its 30th anniversary, the National Organization for Rare Disorders (NORD) has announced ten new grants as part of its Rare Disease Research Grant Program! NORD is the…
According to a story from Pulmonary Hypertension News, a recent study has found that lapses in treatment adherence can result in serious side effects for pulmonary arterial hypertension (PAH) patients…
According to a story from princetoninfo.com, Sara Cooper was diagnosed last November with amyotrophic lateral sclerosis. Sara has had an active career as an event planner, marketing consultant, and real…
According to a story from Global News, Charleigh, the three year old daughter of Trevor Pollack and Jori Fales, was recently diagnosed with CLN2 Batten disease, which is a rare…
According to a story from Cancer Therapy Advisor, a recent study published in the journal Cancer Medicine indicated that chronic lymphocytic leukemia (CLL) patients who dealt with adverse events during treatment…