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).…
According to a story from pharmavoice.com, a new partnership between the biotechnology company Vitrisa Therapeutics and the Foundation Fighting Blindness Clinical Research Institute (FFB-CRI) is in the works in order…
A study has just been published in the New England Journal of Medicine (NEJM) that found that the drug Brineura slows down the deterioration of language and motor function in…
A large clinical study will be carried out to develop a protocol for identifying a rare primary immunodeficiency disease called WHIM syndrome in patients, reports BioPortfolio. The Jeffrey Modell Foundation,…
Phillippa Farrant, whose son, Daniel, has been diagnosed with Duchenne muscular dystrophy (DMD), recently appeared in Parliament in the UK to fight for improved access to care and treatment for…
According to a story from Harvard Health Publishing, a common problem with the upper chambers of the heart which cause the heart to pump inefficiently may be made less severe…
Click to read parts one and two of this story. According to the Center for Disease Control, Lyme disease is the fastest growing insect-borne infectious illness in the United States.…
Doctors haven’t been able to work out the cause of a young woman’s medical symptoms, and now the New York Times is appealing to the public for suggestions. This article…
What do bitcoin and healthcare have in common? Not much until recently. Now an established healthcare provider is launching a system based on patient empowerment. The platform focuses primarily on…
A gene therapy being developed as a treatment for Sanfilippo syndrome has just been awarded Regenerative Medicine Advanced Therapy (RMAT) status in the US, reports GlobalGenes. Sanfilippo syndrome is a…
He was billed as "The 8th Wonder of the World," and this month a new documentary on the legendary WWE wrestler, André the Giant, premiered on cable outlet HBO. According…
A clinical trial that gave children with progeria an experimental drug that was initially intended for cancer patients has produced promising results, reports The Washington Times. The study found that children…
Let's get moving in May! First off, for all you pop culture/Internet meme lovers, it's that time of year... May means we're on the eve of summertime, knee-deep in springtime,…
The inaugural Homocystinuria Network America Conference was held April 21st-22nd in Westford, MA. The families in attendance traveled from all across the US for the event. They all have different…
According to a story from PR Newswire, The Marfan Foundation and Backpack Health are partnering so that the foundation can begin the creation of a patient registry for people affected…
The Australian government will subsidize a new treatment for Hodgkin lymphoma, bringing its price down from $200,000 per course to $39.50 for most people, and $6.40 for concession patients, reports…
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