Each and every day, the medical community (and the world) learn more about COVID-19. What began as a novel coronavirus in Wuhan, China has turned into a global pandemic…
According to a recent press release, EspeRare received Breakthrough Therapy Designation for its investigational therapy ER-004, designed for the prenatal treatment of X-linked hypohidrotic ectodermal dysplasia (XLHED). Ultimately, this…
In some cases, people have a medical condition that doesn't respond well to treatments. The journey to finding an effective treatment can be long and difficult. But for patients…
According to ASH Clinical News, a phase 1 trial of lenzilumab illustrated clinical benefits for approximately one third of participants with chronic myelomonocytic leukemia. It is a novel antibody, and…
When it comes to researching and finding treatments for rare diseases, funding plays a crucial role. Recently, the Medical Research Future Fund (MRFF) Stem Cell Therapies Mission presented Monash…
There are multiple forms of Batten disease, each with their own distinct genetic cause. One of these forms is caused by a mutated CNL6 gene, but researchers were unsure as…
One huge goal throughout the medical world is to find effective and well-tolerated treatments for patients with cancer. According to News Medical, researchers recently discovered one beneficial treatment for…
According to a story from The Boston Herald, a new treatment for acromegaly called Mycapssa was recently approved by the US Food and Drug Adminstration (FDA). Developed by Chiasma, a…
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.
