Many medical insurers follow a practice known as step therapy. A new law presented to legislators in Maine proposes to protect patients from such practices. In her column for Central…
Kelsey is a unique child. Her story still doesn’t have an end or an answer. Only suggestions. Maybe mitochondrial disease. Brain atrophy. The two could even be connected. Kelsey’s parents…
According to a story from the Peterborough Telegraph, James Willis received a disaster diagnosis on his first Valentine's Day after getting married at age 29. James had been worried about…
According to a story from the Sydney Morning Herald, two-year-old William McLennan, son of Naomi Taylor and Ben McLennan, was diagnosed with spinal muscular atrophy about a year ago. The…
According to a story from news-medical.net, medical researchers have discovered that the gene called ari-1 can increase the risk aortic aneurysms. Previously, the precise genetic risk factors were not understood,…
The first patient has just been dosed in a Phase 3 trial of a drug to treat spinal muscular atrophy, reports Benzinga. The drug, called AVXS-101, is a gene therapy…
The UK government is being petitioned to hold a referendum on the decriminalisation of the drug cannabis. This has implications for people with diseases that may be helped by using…
According to a story from philly.com, a recently released set of guidelines is placing new emphasis in the need for patients not to delay treatment during the early stages of…
The National Center for Advancing Translational Sciences (NCATS) supports rare disease patients and their communities. They provide translational research funding, tools and other resources that are helping to address unique…
Acceleron Pharma has just received Fast Track designation from the US Food and Drug Administration (FDA) for their experimental treatment ACE-083 designed to treat patients with facioscapulohumeral muscular dystrophy (FSHD).…
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.
