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…
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