In 2008, Sandra Bedrosian Sermone gave birth to twin boys, Tony and Rocco. Early on, Tony was in and out of the hospital for four years with no diagnosis as he lost the ability to swallow, walk and talk. At 4, he was diagnosed with a number of conditions including...
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Boston, Mass. — Childhood interstitial lung disease (chILD) is a group of rare, genetic lung disorders affecting infants and children who typically present with a range of symptoms, such as shortness of breath, rapid breathing and coughing. While many causal genes have been identified for chILD, understanding its pathogenesis has...
Novato, Calif. – Restricting calories is known to improve health and increase lifespan, but much of how it does so remains a mystery, especially in regard to how it protects the brain. Buck scientists have uncovered a role for a gene called OXR1 that is necessary for the lifespan extension...
Digital communities have become support networks for patients of rare diseases, as well as professionals. They boost access to research, encourage advocacy, and aid evidence-gathering When Lucinda Andrews learned that her newborn son was only the 16th person in the world known to be diagnosed with an ultra-rare genetic disorder, she...
STOCKHOLM, Sweden – Vicore Pharma Holding AB (STO:VICO), unlocking the potential of a novel class of drugs, angiotensin II type 2 receptor agonists (ATRAGs), today announced that Japan’s Ministry of Health, Labor and Welfare (MHLW) has granted Orphan Drug designation to buloxibutid for the treatment of idiopathic pulmonary fibrosis (IPF)....
ITHACA, N.Y. — By studying individuals who spontaneously clear hepatitis C infections, a team of researchers has identified viable vaccine targets for a disease that infects 70 million worldwide with case numbers increasing every year. It turns out that a quarter of people who become infected with the hepatitis C virus...
Coronary artery bypass surgery provides long-term benefits for children whose hearts and blood vessels are damaged by Kawasaki disease, Japanese researchers report in Circulation: Journal of the American Heart Association. Researchers followed 114 people for up to 25 years who had bypass surgery as children or adolescents (ages 1 to...
Tucson, Arizona — Critical Path Institute’s (C-Path) Duchenne Regulatory Science Consortium (D-RSC) is excited to announce the launch of a groundbreaking model-based Clinical Trial Simulator (CTS), specifically designed to improve design of efficacy studies for potential therapies for Duchenne muscular dystrophy (DMD). This pioneering Drug Development Tool is set to...
TUCSON, Ariz. — Critical Path Institute (C-Path), a leader in accelerating drug development for rare diseases, today announced the targeted integration of additional Friedreich’s ataxia (FA) datasets into C-Path’s Rare Disease Cures Accelerator-Data and Analytics Platform (RDCA-DAP®) as part of a partnership with Friedreich’s Ataxia Research Alliance (FARA). This update...
Tucson, Arizona — Critical Path Institute (C-Path) today announced the formation of a new task force under its Rare Disease Cures Accelerator-Data and Analytics Platform (RDCA-DAP®), dedicated to advancing therapeutic development for Progressive Supranuclear Palsy (PSP). This initiative brings together leading organizations and experts in a concerted effort to tackle...