The first ever drug in the United States for treating transthyretin amyloid cardiomyopathy (ATTR-CM) has finally been approved by the FDA. It's amazing news for this patient community. But, you…
According to a story from BioSpace, the drug development company Dynacure recently released an announcement in which the company stated that its application for a Phase 1/2 clinical trial had…
According to a story from forum.facmedicine.com, Dr. Rahul Desikan has been dedicated to the study and research of neurological disease such as amyotrophic lateral sclerosis (ALS). Then, in a cruel…
Cedric Rutland, a pulmonologist at Pacific Pulmonary Medical Group has shared his experience, research, and thoughts on cognitive bias in an effort to spread awareness for the issue, and hopefully,…
According to a story from sciencemag.org, an experimental gene therapy for the rare genetic disorder X-linked myotubular myopathy is starting to produce significant improvements for patients. A devastating disease that…
.jpg)
An article in Globe Newswire quotes National Institute of Health statistics for malaria as being between 300 to 500 million cases worldwide resulting in 1.5 to 2.7 million deaths annually. People living…
Transthyretin-Mediated Amyloidosis Cardiomyopathy The FDA has just approved the very first treatment for hereditary or wild-type transthyretin-mediated amyloidosis cardiomyopathy (ATTR-CM). ATTR-CM is a rare disease that occurs when amyloids, an…
According to a story from KTVZ, Jill Noe and Whitney Bliesner have always shared a special bond. They are twins, now 34 years old. Despite their closeness, they do share…
According to a story from cbc.ca, Lyle Daniels became one of the first patients to try an experimental treatment called Ocrevus for his progressive multiple sclerosis. Just recently, the province…
According to a story from Science Daily, a team of scientists affiliated with UT Southwestern's Children's Medical Center Research Institute are hard at work pioneering an innovative new approach that…
A recent report that was published in the Swiss Medical Weekly outlines data gathered by a Swiss panel of experts on plasma cell myeloma. The report offers an update and…
According to a story from CRWE World, the drug company BioMarin Pharmaceutical, Inc. recently announced that Palynziq, a treatment developed by the company for phenylketonuria, has earned marketing authorization from…
The 2019 Congress of Clinical Rheumatology held May 2nd through the 5th in Destin, Florida featured a talk by Yusuf Yazici, the Director of the Behçet Syndrome Center at the…
According to a story from fox43.com, an experimental therapy for the genetic form of amyotrophic lateral sclerosis (ALS) has been termed "game changing." A study that will soon be presented…
According to a story from EurekAlert!, the biopharmaceutical company Celgene Corporation and the gene therapy company bluebird bio, inc. recently announced that the interim results for a Phase 1 clinical…
Chronic Fatigue Syndrome Chronic fatigue syndrome (CFS) is a rare disease which causes, most notably, persistent fatigue that doesn't improve with rest. It is also sometimes referred to as myalgic…
According to a story from globenewswire.com, the pharmaceutical company Acer Therapeutics recently announced the release of data from a long-term observational study of patients with vascular Ehlers-Danlos syndrome (vEDS). All…
Dutch Bros Coffee is hosting their 13th annual fundraiser for amyotrophic lateral sclerosis (ALS) this Friday, May 10th, 2019. It's called "Drink One for Dane" in honor of Dane Boersma,…
According to a story from Medical Xpress, living with Tourette's syndrome can be a unique challenge, especially for women. The condition, which is characterized by repetitive involuntary movement or vocal…
.jpg)
The News Hub at Washington State University recently carried an article describing initial success by researchers towards developing a drug for a rare form of muscular dystrophy. Several thousand people…
In February, Li Kegiang, Premier of the State Council, announced that the Chinese government was going to take steps to improve rare disease patients access to medicine. As part of…
.jpg)
Researcher: “MND is not an incurable disease. It is an underfunded disease” The Trinity Times recently interviewed Prof. Orla Hardiman on the subject of motor neuron disease (MND), also…
According to a story from finanzen.at, a recent early stage clinical study showed positive results from an innovative stem cell therapy treatment. In the study, this therapy was able to…
What Exactly is Mast Cell Disease According to The Mastocytosis Society (TMS), there are three forms of mast cell disease. All involve the malfunction or over-development of mast cells in…
According to a story from The Intercept, Ady Barkan's life is slowly slipping away as his amyotrophic lateral sclerosis continues to progress. Despite this, Ady has developed a reputation as…
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.
