When the 19-year-old French woman first visited the hospital, she was extremely lethargic. She displayed neurological symptoms. Upon kidney failure, the patient was admitted into the ICU. But soon, doctors…
By Danielle Bradshaw from In The Cloud Copy Expressive language sampling, or ELS, could potentially help researchers better measure how well treatments work for patients with fragile X syndrome or…
Arabel Standingbear sketches figures with lead pencils and paints realistic scenes with acrylics. Her poem was accepted by a museum. She has won in local art contests between schools, and…
Nicox SA, an international ophthalmology company, recently announced a Phase 3 clinical trial for NCX 470 to lower intraocular eye pressure. The article recently appeared in Eye Wire News. The…
Happy Friday! The world is full of difficult news right now. If you're looking for ways to educate yourself on some of the problems facing the world right now, especially…
An estimated 25-30 million Americans are part of the rare disease community. If you're one of them, you share a common (yet uncommon) experience: public scrutiny, difficulties in diagnoses, treatment…
June is recognized as Myasthenia Gravis Awareness Month. Primarily organized by the Myasthenia Gravis Foundation of America (MGFA), this event is focused on spreading awareness as well as boosting efforts…
According to a story from PR Newswire, CureDuchenne Ventures has recently announced the initiation of its CureDuchenne Ventures 2020 Pitch Contest. This marks the first time that the contest has…
According to a story from BioSpace, the radiopharmaceutical company Clarity Pharmaceuticals has recently announced that the US Food and Drug Administration (FDA) has granted Rare Pediatric Disease designation to its…
According to a story from BioSpace, the biopharmaceutical company MacroGenics, Inc. has just announced that the US Food and Drug Administration (FDA) has given Orphan Drug designation to the company's…
Acadia Pharmaceuticals recently released the results of their Phase 3 trial of Nuplazid, a treatment for the psychosis that Parkinson's disease patients experience. This medication has been approved since 2016,…
Gut bacteria has been connected to a number of conditions: depression, diabetes, and now Parkinson's disease. A study published in Annals of Neurology has shown that gut bacteria is linked…
June marks the start of Aphasia Awareness Month, designed to educate many on aphasia: causes, long-term affects, and the lives of those living with this condition. But, as the University…
According to News Medical, a recent international study designed to understand Lesch-Nyhan syndrome and its potential treatments discovered something intriguing: biochemical changes in the skin, cerebrospinal fluid, and urine. Now,…
According to Disability Horizons, an extremely talented filmmaker with muscular dystrophy recently won an Award of Recognition from The Best Shorts Film Competition. Stephanie Castelete-Tyrrell's film, entitled Muscle Warriors, documents…
Stacey Saiontz is many things: a worker, a wife, a mother. But right now, she is concerned. As an allergy parent, or a parent whose child has severe food…
According to a story from PR Newswire, the National Organization for Rare Disorders (NORD) has recently awarded a total of eleven new grant awards that will help fund essential research…
According to a story from EurekAlert!, a recent international study is utilizing data from 45 patients to describe a mostly unknown new rare genetic disorder. The researchers are recommending that…
According to a story from Sarcoidosis News, while the global challenge of the COVID-19 pandemic has been an obstacle for advancement in many rare diseases with trials and research grinding…
According to a recent article in Medscape, the FDA has issued accelerated approval for Bristol-Myers Squibb's Pomalyst (pomalidomide). Pomalyst was approved as the first new oral treatment for AIDS-related…
Ultomiris, a product of Alexion Pharmaceuticals, has recently been studied in a Phase 3 clinical trial. This study was intended to ultomiris' effect on life-threatening complications of atypical hemolytic uremic…
According to a story from Neurology Advisor, a recent study has found that patients living with neuromyelitis optica spectrum disorder (NMOSD) are at an increased risk of experiencing bone loss,…
In a recent article that appeared in Science Magazine, Amy Boland described her twelve-year quest to find a cure for the cancer that invaded her lymph system. At first,…
According to a story from globenewswire.com, the biopharmaceutical company Apellis Pharmaceuticals, Inc. has announced that it is currently making plans to submit a New Drug Application (NDA) to the US…
According to a story from UNC Health Care, the Belgian drug company Argenx recently announced that its investigational treatment efgartigimod has performed well in a phase 3 clinical trial. The…
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