According to a story from BioPharma Dive, the advent of gene therapy development at Spark Therapeutics has been five years in the making. At this juncture, it has been nearly…
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…
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,…
.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 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…
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…
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,…
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…
Uveitis Uveitis is a rare condition in which the uvea of the eye becomes inflamed. The uvea is responsible for providing the majority of the blood to the retina and…
An Unexpected Emergency Kristin Hardy and her sister Margaret are no strangers to rare disease. Kristin has primary progressive multiple sclerosis (MS) and Margaret has relapsing-remitting multiple sclerosis. Margaret also…
Happy Thursday! This week we're highlight a variety of articles. First, we're starting with a strong call-to-action from contributor and advocate, Sharon. Next, we have an article about rare disease…
According to a story from BNN Bloomberg, Novartis AG, a drug company that is nearing the completion of the development of a potentially groundbreaking new gene therapy for spinal muscular…
After 20 agonizing years, a mother and father have finally received answers regarding their daughter's health. It shouldn't take this long. The Diagnosis Their daughter was diagnosed with two very…
Sarcoidosis Sarcoidosis is a rare inflammatory condition that causes granulomas to buildup within the organs. These can cause permanent organ damage. While the condition can affect various organs, and presents…
According to a story from finanzen.ch, the pharmaceutical company Eisai, Inc. announced that that company presented a total of four abstracts which were related to a Phase 3 clinical trial…
Doctor Alan Pestronk has identified a new and very rare autoimmune disease. He is now trying to spread the word about its existence to other doctors in an effort to…
Noonan Syndrome Noonan syndrome (NS) is a rare disorder that is typically diagnosed at birth. It affects the child's development resulting in stunted growth, abnormal facial features, and heart defects,…
According to a story from BioPortfolio, the biotechnology company Tiziana Life Sciences plc recently announced encouraging results from an interim safety review of the company's Phase 2a clinical trial. This…
Doctor Ashutosh Mangalam recently conducted a study examining the affect of Prevotella histicola, or P. histicola, a bacteria found in the human gut, as a treatment for multiple sclerosis (MS). Specifically, he…
According to a story from Healthworld, the government of Kerala, an Indian state located on the southwestern coast, has announced plans to take action in order to treat patients with…
Stevens-Johnson Syndrome Stevens-Johnson Syndrome (SJS), otherwise known as toxic epidermal necrolysis, is a rare disease caused by allergic reactions to common drugs or a reaction to an infection. Some of…
According to a story in the New York Times, cases of measles in the US have surpassed 700 in the year of 2019 so far. Regarded as the most serious…
© 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.
