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…
"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 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…
GlaxoSmithKline (GSK) has just announced that Zejula, otherwise known as niraparib, has just been approved for cancer patients beyond those who have a BRCA+ mutation. The patients now included in…
According to a story from Fabry Disease News, a recent study has found that certain cells tend to age more rapidly in patients with Fabry disease when compared to unaffected…
According to a story from Buzzfeed News, Facebook has been receiving more negative press than ever lately, and so far it often seems justified. From the Cambridge Analytica scandal to…
According to a story from BioSpace, the biopharmaceutical company Newron Pharmaceuticals S.p.A. has announced that is partnering with the Rett syndrome disease community in order to sponsor an all new…
Narcolepsy is a rare neurological disease that affects the sleep cycle. Symptoms include sleep paralysis, excessive daytime sleepiness, disrupted nighttime sleep, hypnagogic hallucinations, and, for around 70% of patients, cataplexy.…
According to a story from Kent Online, like many other four year olds around the world, Olivia Wilkes will start attending school this year. However, her situation is different from…
If you or someone you know has ADPKD, email us at [email protected]. We’d love to hear from you about your experience! According to a story from Medscape, a subject of…
The number of systemic juvenile idiopathic arthritis patients who develop lung disease (sJIA-LD) is rising in both the U.S., Europe, the Middle East, and Canada. This trend has been notable…
According to a story from Express Digest, young mom Emily Nixon, age 25, was heartbroken when her oldest daughter Darcy Roger, age 6, passed away only five days after her…
Little Rozárka’s mother isn’t giving up. Her daughter is the only person in the Czech Republic with tetrasomy X, and is one of 50 people in the world with the…
Results of a study reported in PLOS ONE that was conducted by a team of scientists indicate that the experimental drug mavoglurant improves responsiveness and eye gaze for patients with…
Actus Therapeutics, a privately held portfolio company of AskBio, has recently announced through PRWeb, the initiation of patient dosing in a clinical trial testing its investigational gene therapy ACTUS-101. About Pompe…
Web-based Living With Chronic Illness Support Group 4th Monday of each month This is a peer support group for patients living with chronic, rare diseases. Featuring Alicia Sloan, MSW, LICSW,…
According to a story from BioSpace, the biopharmaceutical company AzuRx BioPharma Inc. recently announced that the first patient has been treated in the company's phase 2 clinical trial. This trial…
According to a story from BioSpace, the US Food and Drug Administration (FDA), has officially approved the drug Trikafta as a treatment for cystic fibrosis, a rare disease that causes…
As originally reported on NPR, Alex Yiu’s mom has a montage of her son's early years. As one watches the videos, you see something: him losing control. He begins to…
When Frankie Avila first got his lung transplant, he was told that, like most organs that are transplanted, his lungs only have a certain amount of time before they…
According to a story from BioSpace, Oncotelic, Inc., recently announced the publication of a study in the Journal of Clinical Research in Pediatrics. This study appears to back up the therapeutic…
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