The approval of Bluebird bio's gene therapy, Skysona, in September 2022 by the FDA marked a significant milestone for treating cerebral adrenoleukodystrophy (CALD), a rare and fatal childhood brain disorder. Skysona's innovation generated hope for families dealing with CALD, yet recent
In a dynamic twist within the pharmaceutical industry, several companies are locked in a fierce race to develop the next generation of obesity drugs. As the battle heats up, each aims to outdo the other, with innovations that promise to revolutionize the way obesity is treated. This race isn't
Oculopharyngeal Muscular Dystrophy (OPMD) is a rare, progressive muscle-wasting disease that significantly impacts patients' quality of life. Limited therapeutic options are available, with current treatments failing to address the underlying muscle weakness driving the disease's
Innovation in biotechnology, particularly within the realm of cell therapy, hinges significantly on the expertise of regulatory professionals. These experts are instrumental in navigating complex regulatory landscapes and ensuring that scientific advances transition into viable, market-ready
The field of spinal muscular atrophy (SMA) treatment has been dynamically evolving, and the latest breakthrough stems from Scholar Rock's Phase 3 clinical trial of apitegromab. This experimental drug is designed to preserve muscle function, representing a potential new standard of care for SMA
The controversy surrounding Cassava Sciences and its experimental Alzheimer's drug, simufilam, has garnered significant attention within the medical and research communities, as well as among regulatory bodies and patient advocacy groups. Allegations of data manipulation and scientific
