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,…
.jpg)
Scientists believe that “it is in our DNA.” According to a recent article in EurekAlert, a five-year study formulated through Open Targets together with the Sanger Institute and their…
According to a press release from Barcelona-based biotechnology company Minoryx Therapeutics, the Company has completed enrollment for a phase 2 clinical study of its experimental Friedreich's ataxia treatment, MIN-102 (generic…
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 BioPortfolio, the drug company Octapharma USA sponsored the 71st Bleeding Disorder Conference, which is put on by the National Hemophilia Foundation. The company is also…
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…
According to a story from MedCity News, interim data from a phase 1/2 clinical trial testing Zolgensma, a gene therapy for spinal muscular atrophy, in patients between the ages of…
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.…
Diagnosis, Treatment, and Adherence There are many factors necessary for an individual to be able to thrive with a serious medical diagnosis. First, they must receive the correct diagnosis so…
According to a recent press release from XBiotech, a Texas-based biotechnology company, patient dosing is now underway in a phase 1 clinical study of the Company's investigational systemic sclerosis treatment, bermekimab.…
According to a story from news-medical.net, the National Institutes of Health (NIH) has doled a total of $31 million on grants this year to 20 different research teams. The goal?…
.png)
A recent article in Forbes highlights a study on diagnostic performance published in The Lancet Digital Health. The study represents the first review to compare the diagnostic accuracy of deep…
According to a story from Medical News Bulletin, a recent study that was first published in the New England Journal of Medicine appears to have revealed a promising new treatment for ulcerative…
According to a story from med.miami.edu, an international research consortium known as Clinical Research in ALS and Related Disorders for Therapeutic Development (CReATe) has just been awarded a renewal grant from…
.jpg)
Mila Makovec began her life as a normal, healthy infant, and according to a recent article in Science Magazine, she continued along this path until reaching the age of…
According to a story from Ankylosing Spondylitis News, a recent study has found that moxibustion, a form of heat therapy, could provide short-term benefits for patients with the rare disease…
A recent press release from Fulcrum Therapeutics, a Massachusetts-based biotechnology company, announced preliminary findings of the company's phase 1 clinical study of losmapimod in the treatment of facioscapulohumeral dystrophy. Losmapimod is…
According to a story from BioSpace, a global collaborative called the Trajectory Analysis Project recently released a study that utilized the North Star Ambulatory Assessment (NSAA), a recently developed measurement…
According to a story from ncbiotech.org, a new partnership between Vertex Pharmaceuticals and RNA-based therapeutics company Ribometrix aims to develop potential treatments for serious diseases, including Huntington's disease, which is…
.jpg)
According to a recent article published by the University of California San Francisco, Pelizaeus-Merzbacher disease (PMD) is a rare neurological disease affecting young boys. PMD fatalities may occur before the child…
According to a recent press release from the Massachusetts-based biotechnology company Avrobio, Inc., the first patient has been dosed in the Company's phase I/II clinical study of their investigational cystinosis…
A Phase 3 trial for Cushing syndrome led by Maria Fleseriu at Oregon Health and Science University has just been completed. It examined a new investigational treatment called levoketoconazole. About the…
Gene therapies have huge potential in the field of rare disease. The fact that these therapies could offer a potential cure otherwise fatal diseases has given families incredible hope. Unfortunately,…
According to a story from the 23andMe Blog, Reni Winter-Evans, who was diagnosed with Parkinson's disease, understands that you don't have to be a scientist to be a big part…
© Copyright 2024 Patient Worthy
Sign Up With a Patient Worthy Account and Share Your Rare Story
- OR -
Make a difference, share your experiences and get paid. Opt-in and join Patient Worthy's panel for paid opportunities such as surveys, market research, patient advisory panels and more.
- OR -
Make a difference, share your experiences and get paid. Opt-in and join Patient Worthy's panel for paid opportunities such as surveys, market research, patient advisory panels and more.
