The FDA has approved a new narcolepsy and cataplexy treatment for pediatric patients! Xyrem is a drug used to control narcolepsy symptoms including cataplexy and excessive daytime sleepiness (EDS). It…
A study has found that more than 80% of participants carrying genes linked to breast cancer didn’t know they had them. The research paper, called ‘Exome Sequencing-Based Screening for BRCA1/2…
When a lot of people think of physical therapy, they think of rehabilitation to improve an injury or some type of ailment. However, the world of physiatry is much broader…
November is Epilepsy Awareness Month! Epilepsy is a central nervous system disorder in which brain activity becomes abnormal, causing seizures or periods of unusual behavior, sensations, and sometimes loss of…
Acer Therapeutics has submitted a New Drug Application to the United States Food and Drug Administration on behalf of Edsivo™, their experimental treatment for vascular Ehlers-Danlos syndrome. To find out…
According to a story from Scleroderma News, many rare disease patients will be heading to the polls on November 6th. Many of them are as deeply concerned about the high…
According to a story from Market Screener, the international pharmaceutical company Recordati recently announced that the European Commission (EC) has recently presented the company's experimental product candidate REC 0545 with…
A study has found that a class of drugs known as MDM2 inhibitors may have the potential to help patients with ocular inflammation uveitis, a serious condition that affects the…
Alnylam has provided updates about their programs for patients with primary hyperoxaluria type 1 (PH1), a rare condition that affects the kidneys and bladder. As part of their Alnylam Act® program,…
Late-onset Pompe disease is associated with many features, one of which is a change to the voice, which may be noticeable in speech and voice quality. To investigate how this…
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.
