As originally reported in Pinnacle Care, when you are diagnosed with a rare disease, it can seem there is the whole world opening that you must learn all the crevices…
According to a story from governmenteuropa.eu, Professor Susanne Kircher, who is associated with the Austrian MPS Society and the Medical University of Vienna, writes about the state of rare diseases…
According to a story from stltoday.com, the Saint Louis Walk for PKD (polycystic kidney disease) took place on Sunday, October 13th, 2019. This event was just one of many that…
According to a story from messenger-inquirer.com, ten year old Abrielle Haynes of Sacramento, CA is often described as a 'china doll' by both family and friends. This is because she…
"It’s like when you suddenly shut off control over your muscles. Your will can’t affect it at all. “I say that I’m a ‘physical schizophrenic,’ and I can only do…
According to a story from pharmanewsdaily.com, the gene therapy company Avrobio has recently announced that the US Food and Drug Administration (FDA) has granted Orphan Drug designation to AVR-RD-02, an…
According to a story from scienceblog.com, a recent study has revealed that large portion of cystic fibrosis cases recorded in the Dominican Republic and Puerto Rico are linked to genetic…
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The concept of N-of-1, or personalization of clinical care, originated in the 1980s according to an article in The Scientist. It began with a sixty-five-year-old Ontario man who was…
According to a story from tmcnet.com, Alkermes plc and Biogen, Inc. have recently announced that the US Food and Drug Administration (FDA) has approved a new treatment for multiple sclerosis.…
According to a story from NBC News, the US Food and Drug Administration (FDA) issued an announcement during the summer detailing a patient death linked to the use of fecal…
According to a story from cancernetwork.com, the experimental drug selumetinib, which is currently licensed to AstraZeneca, has showed positive results in the phase 2 clinical trial. In this study, the…
What started as an outing to a Virginia Tech event blossomed into the mutual love between a young girl with a rare disease and the Virginia Tech sports community (aka…
Recently, different celebrities have been making their chronic conditions public via social media. Lena Dunham was motivated for a similar reason to Billie Eilish, who revealed she has Tourette syndrome,…
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490 words 8% vs 1306 words 3% According to a recent Globe Newswire release, Progenics Pharmaceuticals, an oncology company that specializes in artificial intelligence and targeted therapies, announced results for its Phase…
According to a story from statnews.com, the approval of the new cystic fibrosis drug Trikafta has caused quite a buzz in this rare disease community. While this new drug offers…
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According to a recent article in Biospace, Catalyst Pharmaceutical’s CMS-001 Phase 3 study ended with mixed results. The trial was billed as the first placebo-controlled double-blind study testing amifampridine phosphate (brand…
According to a story from us.acrofan.com, the biopharmaceutical company AstraZeneca recently announced the release of positive results for a clinical trial testing the company's drug durvalumab (marketed as IMFINZI) in…
"We went from being able to treat no patients to being able to treat nearly all patients in seven years." 90% of cystic fibrosis (CF) patients now have a new treatment…
Hogan Teem was a healthy seventeen year old. He had a girlfriend and he spent his summers mowing lawns in his North Carolina hometown. Hogan played football, baseball, and…
According to a story from Medical Xpress, an international team of scientist from 34 different countries are putting their heads together to identify the most effective treatments for ulcerative colitis,…
According to a story from shrewsbury.wickedlocal.com, biomarker testing has now become an important step for the treatment of cancer. In the past, most cancer treatments were based on where the…
As originally reported in NBC News, Mitchel Herndon was witty, sporty, and very close with his siblings. At 19, he began to study as a freshman political science and theology…
According to a story from The Washington Post, data regarding the recently approved drug Trikafta, which is designed to treat cystic fibrosis, was so convincing that the US Food and…
FA Fanconi anemia (FA) is a rare disease in which the bone marrow in the body essentially fails. It is a genetic condition where the body progressively becomes deficient in…
According to a story from newswise.com, biologic medicines have had a major positive impact on outcomes for patients with the rare disease juvenile idiopathic arthritis (JIA). However, as these medicines…
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