According to a story from the Rare Disease Report, the results of a recent survey questionnaire indicate that communicating with other rare disease patients can cause a significant improvement in…
According to a story from Newswise, neurosurgeons at UC San Diego Health can now provide patients with an auditory brainstem implant (ABI) that can preserve some degree of hearing in…
A condition called XLHED that was thought to be incurable has been treated, and a paper describing the procedure, which can be found here, was released on the 26th April…
Matt Millen played linebacker in the NFL for 12 seasons. He was a tough competitor and won 3 Super Bowl rings. For eight years, he was the General Manager of…
Narcolepsy is a rare disease subject to many misperceptions and false assumptions. All of that misinformation belies the fact that narcolepsy is a serious neurological disorder, with symptoms that can…
According to a story from bioportfolio.com, a recent study has demonstrated the effectiveness of Trelegy Ellipta, a unique three component combination treatment, in reducing the effects of chronic obstructive pulmonary…
Every year, May 20th is celebrated as International Clinical Trials Day. On this day organizations that are involved in the fields of science and medicine hold events to promote education…
Back in February, we covered the $1,000,000 IPF Catalyst Challenge. Announced in April, innovators from around the world submitted meaningful solutions to improve the quality of life of those living with…
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…
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