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…
As a second year pre-medical student at Northeastern University, my desire to become a healthcare professional flourishes every day. An advantage of being a student who is just beginning to…
Niemann-Pick disease type C (NPC) is a rare disease caused by a defected NPC protein. In a healthy individual, this protein helps the body process cholesterol. Those diagnosed with NPC experience…
According to a story from the Borehamwood & Elstree Times, the family of 11 year old girl Sivan Hermon is plagued by a rare disease: primary pulmonary hypertension. As the…
Teresa Scruton is not a physican nor a nutritionist but she has been a person with hereditary angioedema (HAE) as well as a researcher for many years. Below she shares…
Hereditary Angiodema Hereditary angioedema (HAE) is a rare disease typically caused by a SERPING1 gene mutation. This protein is responsible for coding for the protein C1-INH. With improper levels of…
According to a story from NBC News, Mitchell Herndon, a 19 year old from Missouri, recently passed away due to the progression of a rare genetic disease that is so…
As originally reported in WWVA, West Virginian Sib Weatherford spent one afternoon this fall doing what nobody though possible: he slid 400 feet down the New River Gorge's high-line in…
About HAE Hereditary angioedema (HAE) is a rare disease that is caused by the deficiency or reduction of a protein called C1-INH in the body. When this protein is not present,…
As originally reported in SF Gate, Jeannette and Victor Vega quickly realized that taking care of their second daughter, Tiana, was not going to be so easy this time. From…
According to a story from Healio, the results from a recent phase 2A clinical trial have demonstrated the effectiveness of an experimental drug in treating congenital adrenal hyperplasia (CAH). The…
According to a story from Technology Review, the story of Mila Makovec shows the potential impact that personalized medicine could have on patients while also revealing some of its limitations.…
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Lately, there have been many newsworthy articles about Novartis and Zolgensma (onasemnogene abeparvovec-xioi). In May 2019 the FDA approved Zolgensma, a one-time treatment for the most severe form of…
BrainStorm Cell Therapeutics has just announced that they have completed enrollment in their Phase 3 clinical trial for amyotrophic lateral sclerosis (ALS). Enrollment was initiated way back in October of…
According to a story from Venture Beat, the company Healx has succesfully raised $56 million in funds that will go towards using artificial intelligence (AI) to identify new potential treatments…
Jessica Walton described the staff at Queen Elizabeth Hospital in King’s Lynn as being in “a bit of a panic” when they thought that she was having a stroke.…
According to a story from PR Newswire, the nonprofit groups Duchenne UK and Parent Project Muscular Dystrophy (PPMD) have jointly awarded grant funding to the tune of $200,000 to Dr.…
According to a story from the TGen Blog, a simple typing mistake lead to a diagnosis for a boy with an incredibly rare genetic disorder. In fact, this disorder is…
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