According to a story from watoday.com.au, Reginald Whiu, aged 73, had been complaining about back pain and was diagnosed with degeneration of the spine by his doctor. This was at…
According to an article from Righting Injustice, a recent study suggests that TNF inhibitors may increase the risk of rare cancers in pediatric patients. The study recommends that children that…
An article from SRN News is reporting that a lot of people with familial hypercholesterolemia are going through life without getting the treatments that could help them, such as statins.…
The EveryLife Foundation for Rare Diseases hosted a panel discussion on Right to Try Proposals, as part of their 2018 Legislative Program for Rare Disease Week on Capitol Hill. What…
A pre-clinical study investigating the effects of antioxidants on mitochondrial diseases has shown promising results, reports EurekAlert. Out of the seven antioxidants trialled, at least two (vitamin E and N-acetylcysteine)…
According to a story from triblive.com, Roger Ziegler has self-published a book about his family's experience with his son Noah. Noah is now a healthy 24-year-old, but there were multiple…
Two-year-old Jeremiah Nelson, born with a condition called spina bifida, has very limited mobility. However, a Michigan high school’s robotics team has decided to help the toddler get moving. Forgoing…
According to a story from the Lancashire Telegraph, Dr. John Timms, age 48, was recently diagnosed with a rare and deadly form of cancer: mesothelioma. Dr. Timms had committed much…
A gene therapy for Pompe disease is the first treatment of its kind to be clinically tested, reports Duke University. Researchers at the University, working with NCATS Therapeutics for Rare…
According to a story from biopharmadive.com, the Swiss pharmaceutical company Novartis has entered a partnership with researchers from the engineering and applied sciences school at Harvard University. The team will…
A team of researchers led by Prof. Michael N. Hall from Biozentrum, University of Basel, have discovered a tumour suppressor protein called LHPP. Their paper, published in the science journal…
Neurologists working at the NYU School of Medicine have developed a test that can identify some rare degenerative brain diseases, reports NYU Langone Health. Published in the Annals of Neurology…
Scientists and many people affected by mitochondrial DNA diseases have been campaigning for legalisation of mitochondrial replacement therapy (MRT) in Canada, reports CBC. It is already legal in several other…
An advocacy group for people with Pompe disease in New Zealand has criticised the government for failing to uphold an election pledge, reports Scoop Independent News. Candidates from all major…
A new drug being developed to treat Angelman syndrome (AS) has just been approved for ‘orphan drug’ status in the U.S., reports GlobeNewswire. The drug, called GTX-101, works by activating the…
One of the greatest gifts of being a kid is imagination. When you're a kid, anything is possible and even the hardest times can turn into something so much better…
For so many in the rare disease community, courage isn't a just a virtue - it's a necessity. Many have to overcome odds and obstacles that most others don't have…
According to a story from curetoday.com, a recent study indicated that many patients with uveal melanoma reported having unmet psychological and informational needs, particularly in the first several months after…
Southampton General Hospital has appointed the UK’s first specialist nurse to give treatment and advice to those suffering from mesothelioma, reports the Southern Daily Echo. Mesothelioma, also known as asbestos…
The average American might not be interested in walking two miles every day. However, for Patricia Coleman, it is an activity in which she proudly partakes! In honor of Sarcoidosis…
Ashley Moore survived cancer. For many people, that’s enough. Moore, however, chose to give back in a surprisingly direct and immediate way. She decided to become a nurse. Keep reading…
A new study conducted at Binghamton University demonstrated that pancreatic cancer cells can be killed with a dual thermal ablation process, which is the combination of freezing and heating, reports…
We hope that everybody is having a great Easter, Passover, or first taste of spring! This week we have a story about sisters with Batten disease who are finally being…
La Jolla Pharmaceutical recently announced a new clinical trial targeting thalassemia. The trial, described as pivotal, investigates a drug referred to as LJPC-401. The treatment is intended to help patients…
As we close out the month of March - it's important to give a little thought to some of the lesser-known rare diseases that are often overlooked. Bleeding disorders is…
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