On Friday, internationally renowned singer-songwriter Sia tweeted, "Hey, I'm suffering with chronic pain, a neurological disease, ehlers danlos and I just wanted to say to those of you suffering from…
According to a story from news-medical.net, a team of researchers have developed a potential new treatment for polycystic kidney disease (PKD), a rare disorder that ultimately causes organ failure. The…
According to a story from derbytelegraph.co.uk, four year old Michal Myslicki, who was born with Batten disease, will soon get access to a critical treatment that will prolong his life.…
Physician and investigator John Wood, MD, Ph.D. of Childrens Hospital of Los Angeles specializes in studying the challenges facing SCD patients. He has currently focused his attention on how oxygen delivery…
According to a story from pmlive.com, the gene therapy company Bluebird Bio's latest recently released results from the company's phase 2/3 clinical trial demonstrates further encouraging data for its Lenti-D…
According to a story from the Cystic Fibrosis Foundation's Community Blog, the internet allows for access to a trove of knowledge and information literally at your fingertips. This had naturally…
NASH Nonalcoholic Steatohepatitis (NASH) is a rare disease in which hepatic inflammation and injury to the cells occur as a result of steatosis (hepatic fat accumulation). Severe cases of NASH…
According to a story from 10tv.com, Kelly Maynard could be hear cheering loudly from the sidelines as her son Jackson, aged 12, scored a touchdown in one of his middle…
.jpg)
According to a recent article in SMA News Today, risdiplam (formerly RG7916) is in ongoing studies of patients with spinal muscular atrophy (SMA). The drug is an investigational medicine developed by Genentech…
According to a story from Biotech 365, the immuno-oncology company Mateon Therapeutics Inc. has recently announced that the company's investigational product candidate OT101 has earned the US Food and Drug…
‘The future is here, it’s just not very evenly distributed’ William Gibson These days there’s much more literature about mal de debarquement syndrome (MdDS) and this resource gives excellent information…
According to a story from Express Digest, four year old Khloe Land of Oregon stepped up to the plate to help her brother get a critical treatment that could very…
I am a 63 widow, with 2 grown kids. I have pure autonomic failure, which you can read more about here. I went on disability in 2004; it took 4…
Prevalence of MS in Korea In South Korea, rates of multiple sclerosis (MS) are generally low. This is because the disease is most prevalent in areas where there is lower sun…
By: Teresa Scruton Hereditary Angiodema Abdominal Attacks There are 3 types of Hereditary Angiodema (HAE) abdominal attacks: Lower abdominal - swelling in wall of colon (large bowel), that causes the…
PROMISE is a huge screening study for multiple myeloma that will invite 50,000 participants who have a higher likelihood of developing multiple myeloma. Dr. Irene Ghobrial of the Dana Farber…
Living life as a rare individual and patient has its challenges day to day. The best feeling in the world, though, is having support: being around people who know what…
“I have cried a lot more in the last ten years of my life than ever before.” A parental odyssey of rare disease, in two of their three children, and…
According to a story from Wales Online, Samantha Josey's childhood was characterized by unpleasant symptoms and health problems, resulting in hospital visits and bouts of serious illness. Her symptoms included…
According to a story from rekordmoot.co.za, the du Plessis family of Moot, South Africa couldn't believe it when two year old Barry du Plessis was diagnosed with Dravet syndrome, a…
According to a story from The Sunday Post, it is an interesting time to be a cystic fibrosis patient in the UK these days. Just weeks after The Scottish Medicines…
Maybe you remember the day of your diagnosis: shocking, confusing, chaotic. Realizing the news will change your life, but not quite knowing how. Jessie Ace was 22 years old, finishing…
As originally reported in Cambridge Independent, inflammatory bowel diseases are about to get a boost in research after Health Data Research UK has awarded a £5 million grant to a…
.jpg)
Waukesha Wisconsin Police Captain Dan Baumann recently held a news conference that was described in an article published in Scientific American. Captain Baumann said that parents of a teenage boy showed…
A recent publication from the US Food and Drug Administration announced the regulatory authority's approval of Nourianz (generic name istradefylline) for Parkinson's patients during "off" episodes. "Off" episodes are windows…
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.
