According to a story from PR Newswire, the National Institutes of Health (NIH) and Three Lakes Partners have awarded a grant to the tune of $22 million to the Pulmonary…
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Stéphanie Tomé is an investigator at the Sorbonne Université in Paris, France. A recent article in PacBio describes Tomé’s research into a disease that becomes progressively worse with each generation.…
According to a story from Parkinson's News Today, the results of a global, online survey appear to indicate that genetically based, inherited Parkinson's disease is actually more common than the…
Elena Frid is neurologist and Lyme disease specialist. She lives in New York City. After opening her neurology practice in Manhattan, she quickly became aware of some huge issues many…
LETHBRIDGE — A Taber, Alta., man is calling on government to take action and help grant Canadians with cystic fibrosis better access to breakthrough treatments. Marten Devlieger was diagnosed with…
According to a story from PR Newswire, the clinical-stage company Vyriad, Inc. and the biotechnology company Regeneron Pharmaceuticals, Inc. are partnering in a licensing agreement and research collaboration to investigate…
As originally reported in Medical Xpress, the ’smart shirt’ is a comfortable shirt for daily life that also functions as wearable technology. The shirt is equipped with sensors that track…
According to a story from WBUR, a preliminary evaluation of a young patients diagnosed with Tay-Sachs disease appears to show some encouraging signs. Two patients were treated with an experimental…
Firdapse, or amifampridine phosphate, is a medication currently approved for Lambert-Eaton myasthenic syndrome, or LEMS. However, Catalyst Pharmaceuticals has been working to evaluate if it can aid other neurological and…
According to a story from Mirror Online, 29 year old Jessica Walton of Cambridgeshire, England, was first diagnosed with the rare disease intracranial hypertension four years ago. Since then, her…
According to a story from Medscape, the drug burosumab (marketed as Crysvita) demonstrated its ability to provide long term benefits for nearly two years to patients with the rare disease…
Happy Thursday, Friends! Welcome to November! This week we're highlighting stories two stories from rare patients in the Czech Republic. We also have a story about two girls in South…
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A Colombian family with more than six thousand living family members was the focus of researchers at several institutes in the U.S. and Columbia. According to an article published by…
According to a story from Targeted Oncology, the US Food and Drug Administration (FDA) has granted Fast Track designation to the experimental drug OMP-305B83, also known as navicixiumab. This designation…
According to a story from abc.net.au, parents Kellee and Jamie Clarkson of Queensland, Australia are calling for the implementation of newborn screening in the state for the rare disorder spinal…
Doctor Leah Kaminsky discusses a new era in healthcare in her recent article that offers hope through new technology. How Can AI Help? AI will be able to predict changes to…
In a story for Hemophilia News Today, author Shellye Horowitz has written a piece titled "An Open Letter to Providers Treating Women with Hemophilia." Women still face discrimination and unequal…
According to a story from abc30.com, Tawny Drain, now 33 years old, was in the sixth grade when she first started having symptoms of a rare illness called Caroli disease.…
Not just anyone can cure their own illness. Understanding how to cure a rare disease is complex, as these illnesses often have unique and poorly understood characteristics. Treatment and diagnosis…
According to a story from Nature, Dr. Carlos Heras-Palou was diagnosed with hATTR amyloidosis when he was just 39 years old. Initially, the devastating diagnosis was completely overwhelming, as the disease…
According to a story from the Children's Organ Transplant Association, the Fitzmaurice family of St. Louis, Missouri will have a ton to be thankful for as the holidays roll around…
According to a story from Daily Nation, Beneddettah Wangui of Kenya had been experiencing chronic pain since her teen years when she was finally diagnosed with fibromyalgia in 2012. However,…
Data in the Modern Age The depth of information available today is unique to our age of technology. Current advances provide a platform to consolidate information on a wide array…
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A recent article in the Washington Post tells the story of a newly-approved cystic fibrosis drug that represents thirty years of scientific work and dedication. The defective gene that causes cystic…
According to a story from Medical Xpress, a team of researchers affiliated with the University of Granada and the research organization CIBERFES have discovered a pair of new mutations linked…
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