Tom Kean, the Senate Republican Majority Leader in New Jersey, is leading the charge to fully realize a recently partially approved bill, which would create a New Jersey Rare Disease…
According to Epidermolysis Bullosa News, staph infections are increasingly common in the wounds or blisters of patients with epidermolysis bullosa (EB). In fact, an analysis of these lesions showed that most had…
Sometimes it's hard to know why you're not sleeping well. Is it because you're nervous for tomorrow’s meeting, or from that impulsive 2PM cup of coffee? For people with Parkinson’s,…
In early February, global biopharmaceutical company Sanofi and biotechnology company Regeneron Pharmaceuticals, Inc. ("Regeneron") shared that its dually developed drug Libtayo (cemiplimab-rwlc) recently received FDA approval. Now, Libtayo stands as…
Biotech company Ovid has released disappointing results from a trial of OV101, a potential treatment for Angelman's syndrome. The Phase 3 trial demonstrated that there was no significant difference in…
Recent research has discovered a new, potential treatment option that could provide an early intervention for Beckwith-Wiedmann syndrome and Russell-Silver syndrome. Published in Cell Reports, this study could provide the…
Recently, biopharmaceutical company GenSight Biologics ("GenSight") announced the publication of data from a study evaluating GS030-Drug Products (GS030-DP or "GS030") as a potential treatment option for retinitis pigmentosa (RP). Although…
Genetic testing is being used more frequently to screen people for the hereditary risk of cancer, according to MedCity News. While advancements have been made and research is ongoing, there…
There are many forms of birth control available: oral contraceptives (birth control pills), intrauterine devices (IUDs), the implant or patch, condoms. But according to Cancer Therapy Advisor, a new study…
Alterity Therapeutics has recently announced the the Michael J. Fox Foundation will be funding a dose optimization trial for ATH434. According to BioSpace, this grant is the second from the…
In early February, pharmaceutical company Ellodi Pharmaceuticals ("Ellodi") shared that its drug candidate, APT-1011, received the FDA's Fast Track designation. APT-1011, or fluticasone propionate oral disintegrating tablet, is designed to…
I used to love a margarita on the rocks with a twist. At some point in my late 20s, I went from the carefree life of a young healthy person…
424 words (source) vs. 431 words (mine) - 3% match Earlier this week, Guinea, a country located in West Africa, reported new cases of Ebola, a rare and deadly infection.…
Dr. Oliver Dorigo of the Stanford University Medical Center was the medical source for an article featured in Oncology. Dr. Dorigo explained that if a woman with ovarian cancer has…
By Tom Seaman If you are like me and probably most people, in today’s busy world it is a big challenge to be still and mentally at ease. This is…
Welcome to the Rare Classroom, a new series from Patient Worthy. Rare Classroom is designed for the curious reader who wants to get informed on some of the rarest, most…
Clinical stage pharmaceutical company KalVista Pharmaceuticals ("KalVista") saw positive data from a Phase 2 clinical trial evaluating its experimental therapy KVD900, shared MedCity News. The Phase 2 trial was exploring…
Genethon and WhiteLab Genomics have entered into a partnership agreement, which will allow them to further develop their work in artificial intelligence and gene therapy. Within the terms of the…
Scientists are researching Alzheimer’s disease (AD) from all angles and gradually closing in on the cause of the disease. According to an article in Neuroscience News, a discovery by researchers…
Learning about rare diseases is one of the first steps towards advocacy. If you or a loved one are affected by cold agglutinin disease (CAD), you may want to get…
The first patient has been dosed in a Phase 1 clinical trial, which is exploring Curis Inc.'s CA-4948 in conjunction with ibrutinib for hematologic malignancies. Examples of hematologic malignancies include…
11-year-old Oliver Shultz is recovering at home after two weeks of stress and fear. It began with an ache in his back, followed by a fever. Soon, the family was…
As shared in a press release by the American Academy of Neurology; the medical community had taken note of the rare brain pressure disorder idiopathic intracranial hypertension (IIH) when they…
According to BioSpace, Homology Medicines has released the first round of data from the clinical trials of HMI-203, a gene therapy being developed for the treatment of Hunter syndrome, also…
According to Charcot-Marie-Tooth News, researchers have discovered a new subtype of Charcot-Marie-Tooth disease (CMT) linked to the sorbitol dehydrogenase (SORD) gene. Read the full study findings published in Nature Genetics. SORD Gene…
© 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.
