Sometimes, rare diseases are so rare that they are just as unique as the individual who has it. One child was suffering from a fatal neurodegenerative disease, and the family…
This week, many cities and regions across the country acknowledged Indigenous Peoples' Day, a day to remember and honor the histories and cultures of Native Americans. The treatment of Native…
The 2019 Global Genes Rare Patient Advocacy Summit was filled with incredible presentations that highlighted steps for progress in research and advocacy organization, provided solidarity for the rare community, and…
As originally reported by EurekAlert, eosinophilic gastrointestinal disorder research will receive a boost with the renewal of a $7.57 million grant administered over five years awarded to the Cincinnati Children’s…
A new joint clinicial investigation has come out with a reminder to patients with primary hyperoxaluria type 2 (PH2) to adherr to the requirements regarding careful follow up proceedings. This…
According to a story from curetoday.com, Betty Strommer had been a breast cancer survivor for years when, in 2013, she was suddenly diagnosed with cancer again. This time, it was…
Gene therapies have huge potential in the field of rare disease. The fact that these therapies could offer a potential cure otherwise fatal diseases has given families incredible hope. Unfortunately,…
According to a story from offspring.lifehacker.com, medical abnormalities and conditions that affect the skin are often something that many of us will notice on our own. Once a skin problem…
A New Collaboration The National Hemophilia Foundation (NHF) is a nonprofit specifically focused on accelerating research for Hemophilia, a rare blood disorder. It was established in 1948 and now has…
Happy October! This week, we're sharing an article on a PKD treatment update. We also have news about a famous singer who shared her rare disease story recently, a piece…
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…
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 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…
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…
“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…
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…
The Center for Chronic Illness is a nonprofit group that is based out of Seattle, Washington. The goals of this organization include the provision of support and educational resources to…
With advocacy organizations gaining traction, sometimes patients with rare diseases may have a handful of options to choose from. Some patients may be quick to join patient advocacy groups…
According to a story from The Guardian, Jeremy Corbyn, the leader of the UK Labour Party, announced a remarkable plan at the most recent Labour party conference that has the…
Innovation and Collaboration Macular telangiectasia type 2 (MacTel) is a rare eye disease that causes progressive vision loss as patients age. Paul Bernstein is a physician at the John A.…
According to a story from Financial Buzz, the biotherapeutics company Bellerophon Therapeutics Inc. has recently announced that it has earned Orphan Drug designation from the US Food and Drug Administration…
Consent is a concept back under discussion in modern conversations as we readdress what it means to cross the line. Consent is no longer seen in the black and white…
According to a story from Spectrum News a recent study revealed two potential therapies that could treat symptoms associated with Rett syndrome, a rare disorder. The study involved the creation…
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