RGX-121 was rejected in early February, with Regenxbio listing the primary reasons given by the FDA as concerns about the ability to properly define a patient population, the use of a natural history ...

A Carrboro mother has pulled in close with UNC researchers after watching her 15-year-old son lose basic skills to a devastating genetic disease. Over several years, Cole regressed, losing most of his ...

The FDA last October paused Intellia Therapeutics’ late-stage CRISPR studies after detecting life-threatening enzyme ...

The FDA has liberated the second of two Intellia Therapeutics clinical trials that were put on hold last October following a report of serious liver toxicity that later led to death.

The approval follows PEGASUS Phase III trial assessing Palynziq efficacy and safety compared to diet alone in adolescents ...

Despite the availability of effective antihypertensive therapies, global blood pressure control rates remain unacceptably low. Contributing factors, such as low treatment adherence, therapeutic ...

Maharashtra BJP MP Anil Sukheorao Bonde has called on the government to ensure continuous life-saving Enzyme Replacement Therapy (ERT) for children with rare diseases. This plea was made during the ...

Xanthine oxidase inhibitors, primarily allopurinol and febuxostat, are the mainstay of treatment. The goal of chronic gout management includes lowering serum urate levels to below 6 mg/dL. 5 Overall, ...

"Life is taken away from these children before they even have a chance to live it," says Megan Kempf of her two children, who have Sanfilippo syndrome ElijahxEmily Photography / SWNS Megan Kempf and ...

Obesity is a global epidemic and a major cause of morbidity and mortality because it increases the risk for comorbidities, including heart disease and fatty liver disease (MASLD). Rates of these ...

Jillian Kubala, MS, is a registered dietitian based in Westhampton, NY. Jillian uses a unique and personalized approach to help her clients achieve optimal wellness through nutrition and lifestyle ...