A story from Charcot-Marie-Tooth News highlights the necessity for genetic testing and a physical examination prior to chemotherapy in order to prevent nerve damage in people with asymptomatic Charcot-Marie-Tooth disease…
In an twist of fate, a brand new gene therapy for the treatment of Adrenoleukodystrophy (ALD) uses one of medicine's greatest nemesis, HIV, to treat this fatal genetic brain disease-- and…
According to a story from SMA News Today, a recent study has demonstrated that the parents of children with spinal muscular atrophy are often skeptical about enrolling their kids in…
Clinical trials of a potential gene therapy treatment for Duchenne muscular dystrophy (DMD) have been put on hold after concerns emerged about the safety of the procedure, reports Genetic Engineering…
Discussion on Right to Try legislation continues, and the federal government could soon change existing rules. Many people wonder whether or not patients should have the right to try potentially…
While learning about cancer at a molecular level while being a medical student, Omar Salman from Virginia Tech Carillon School of Medicine learned much more firsthand when he was diagnosed…
According to a story from Wave Newspapers, Danielle Guinn, a woman diagnosed with cerebral palsy and epilepsy, is not letting her disability slow her down, and is currently participating in…
According to a story from asianscientist.com, a group of Japanese researchers have managed to unearth a genetic mutation that is associated with progressive supranuclear palsy. The discovery is helping scientists…
Researchers at the University of South Florida have developed a blood test that may improve diagnosis for many diseases, including HIV and Lyme disease, reports EurekAlert. The new test uses…
Donna Keck has multiple sclerosis (MS). She's 61-years-old and has lived a life full of health complications, reports Chicago Tribune. Nevertheless, Keck battled through these challenges and eventually founded a grounded…
Patient Worthy’s partner, The MDS Foundation, will be hosting another wonderful event for MDS patients and caregiver. Event Summary: Whether you are a newly diagnosed patient, a long-term survivor, or a…
Cindy Rogers never gets a day off. She barely recalls a time when she slept for more than a handful of consecutive hours. This is because Cindy serves as primary…
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…
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