According to a story from Juvenile Arthritis News, a recent study from China has found that early treatment with infliximab (marketed as Remicade) can reduce inflammation and indicators of active…
According to a story from BioSpace, the genetic medicines company Homology Medicines, Inc., has recently released preclinical data supporting the development of two experimental gene therapies. One, called HMI-202, is…
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…
As originally reported in South Africa Latest News, Healx Ltd. has raised $56 million dollars to fund their development of treatment options for rare diseases using artificial intelligence. The British…
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Over the years Alzheimer’s patients and their families have seen more than one hundred twenty drugs that were developed for the treatment of Alzheimer's have failed. For a while,…
As originally reported in Michigan Medicine News, the Nephrotic Syndrome Study Network (NEPTUNE) has received their third five year cycle of funding to research rare kidney diseases. Right now, NEPTUNE…
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…
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A guest speaker at the 2019 Rare Disease Forum recently held at the North Carolina Biotechnology Center acknowledged that screening is necessary to justify the need for a particular…
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…
According to a story from globenewswire.com, the experimental drug somatrogon, which is being co-developed by Pfizer, Inc. and OPKO Health, Inc., was able to achieve its primary endpoint in a…
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…
The Gap Tysabri is a therapy that has shown efficacy for reducing disease activity in patients diagnosed with pediatric-onset multiple sclerosis (POMS). Previous investigations have indicated that up to 58%…
In a story from Medical Xpress, the authors of a recent study declared that maternity care for pregnant women with Ehlers-Danlos syndrome (EDS) is in dire need of improvement. Ehlers-Danlos…
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 MDMag, the US Food and Drug Administration (FDA) has recently approved a label expansion for the drug treprostinil (marketed as Orentiram). This update to the…
When Jordan Berkhoudkt was in middle school, he began to have pain that they couldn’t find a way to relieve. He couldn’t get an accurate diagnosis from the doctors in…
CNN recently covered a story in the publication Nature about a paper explaining base editing (or prime editing). The researchers who created the technology set forth the process of using base…
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…
Happy Thursday! This week we're highlighting four patient stories: a woman managing her HAE symptoms, a singer with a scary diagnosis, a Bechet patient building support networks, and a CIDP…
According to a story from Pulmonary Hypertension News, pulmonary arterial hypertension and its variants can ultimately result in a cascade of impacts on other areas of the body, including the…
According to a story from irishmirror.ie, Danny O'Donoghue, the vocalist and keyboard player for the Irish rock band The Script, recently talked publicly about his struggle with Wilson's disease, a…
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