For many, being diagnosed with any disease – let alone a rare disease – is understandably devastating. Many have described managing their new lives as learning a new language or…
Researchers are arguing that the certain drugs, including the erectile dysfunction drug Viagra, could be used as cancer treatments, reports MedicalXpress. This theory is based on a range of pre-clinical…
A recent study, published in Nature Communications and reported on by the BBC, has discovered five genes that are linked to pulmonary arterial hypertension. Pulmonary arterial hypertension (PAH) is the…
The 2018 Mrs. Maine International Pageant will be so much more than a celebration of poise and beauty. It will also make history when Devan Demmons will proudly walk on…
The pharmaceutical company Lonza has just opened a new state-of-the-art manufacturing facility to produce cell and gene therapies. You can find the full press release here, on Lonza’s website.…
According to a story from Summit Medical Group, a recent study indicates that survivors of testicular cancer may have a greater risk for heart disease. Compared to many types of…
According to a story from stv.tv, 14-year-old Kira Noble has been fighting against neuroblastoma for around over four years. She first began to realize that something was wrong when her…
According to a story from wired.com, a new genomics marketplace called Helix will soon start selling genetic tests directly to consumers. These tests are the same ones that doctors use,…
According to a story from globenewswire.com, the pharmaceutical companies Ultragenyx and Kyowa Kirin recently issued an announcement that the U.S. FDA has granted approval to their new treatment Crysvita (burosumab-twza).…
According to a story from news.vanderbilt.edu, Charlotte Haffner has been working hard to advocating on behalf of amyloidosis patients. She recently underwent a heart transplant and stem cell transplant in…
Researchers have found that forms of certain genes can influence how successful a blood and marrow transplant is, reports Roswell Park. This has important implications for understanding patient outcomes and…
As a female Bible college student, an outside marker of pious and godly womanhood consisted of having a male significant other. While this is not strictly a religious sentimentality about…
It's a parent's worst nightmare-- a rare disease that strikes early in life, and without treatment, can cause a patient's death. Tyrosinemia type 1 (TYR1), is a form of a…
According to a story from Charcot Marie Tooth News, a small subdivision of the National Institutes of Health (NIH) is leaning into its role to assist with research into rare…
According to a story from PR Newswire, the pharmaceutical company Therapix Biosciences recently announced the results of a Phase IIa study for its drug candidate THX-110. The therapy is indicated…
According to a story from medpagetoday.com, researchers are beginning to realize that symptoms of pediatric spondyloarthritis present themselves in a distinct way when compared to the symptoms that are experienced…
It is international Pompe Day! “Together we are Strong” Today is meant to help create awareness for the Lysosomal Storage Disorder Pompe Disease, which is both rare and underdiagnosed. Pompe…
According to a story from femalefirst.co.uk, Jas Shaw of the electronic music project Simian Mobile Disco was recently diagnosed with AL amyloidosis. The group was planning a tour of the…
Hope everybody is enjoying the warm weather! This week we have patient stories about a woman with MS who started a support group and a rare mom who took issues…
According to a story from QQ Malaysia Online, patient advocates are looking for ways to raise awareness of rare diseases in Malaysia and get help to forgotten patients that desperately…
According to a story from PR Newswire, a recent survey suggests that a greater number of U.S. citizens know about the existence of pancreatic cancer than ever before. This survey…
According to a story from PR Newswire, the science and tech company Merck recently announced that Mavenclad (cladribine) was recently approved for public use by the UAE Ministry of Health…
Now these are the kind of stories we love to read about! Canadian Revée Agyepong is the first adult in Canada to be cured of sickle cell anemia through stem cell…
According to a story from scimex.org, a recent data review is calling into question the previously held consensus that common pain medicines such as NSAIDs and paracetamol could make inflammatory…
No matter which way you spin it, Ehlers-Danlos syndrome (EDS) is generally not a great time. EDS is a rare genetic condition that affects connective tissue throughout the body. It…
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