Learn how caregivers can care for themselves so they can better care for others. Science & Innovation A New Road to Migraine Relief Migraine, a debilitating neurological disorder, affects over a billion people globally. The cause of migraine remains an active area of research. Purpose & Idea...
As challenges and increased costs continued this year for clinical trials due to the COVID-19 pandemic, the FDA remains committed to supporting rare disease research by providing existing grantees with additional funding. These resources allow ongoing studies to implement ...
In this neurodegenerative disease, a progressive death of retinal ganglion cells (RGC)... R Herrero‐Vanrell - 《Acta Ophthalmologica》 被引量: 0发表: 2024年 Guidance of Medical Care for Familial Exudative Vitreoretinopathy:Research on Rare and Intractable Diseases, Health and Labour Sciences ...
Reports on several health research grants in the U.S. Clinical trials on the safety and efficacy of products for rare diseases and conditions; Cooperative agreement applications to support faculty positions in medical schools specializing in training related to sexually transmitted disease prevention and...
Sarepta provides grant funding for programs that enable education, address unmet needs of rare disease patients, promote the highest standards of care, raise awareness of ongoing research, or focus on other critical community initiatives. Requests will be considered across the spectrum of neuromuscular...
The FDA granted this application Fast Track and Priority Review designations. Vyondys 53 also received Orphan Drug designation, which provides incentives to assist and encourage the development of drugs for rare diseases. In addition, the manufacturer received a rare ...
Further, WP1066 can enhance the antitumor effects of the immune system and its response to tumor activity. Research also suggests the agent may have a dual-action mechanism that allows for the treatment of an array of resistant tumors and can be paired in novel combinations for unresponsive ...
The US Food and Drug Administration (FDA) has granted accelerated approval for PTC Therapeutics’ KEBILIDI, the first gene therapy in the US that is directly delivered to the brain.
Since Luke’s diagnosis, we have become deeply connected to the rare disease community. While the FDA recognizes more than 7,000 rare diseases, many receive little attention or funding for research, leaving families like ours with...
high-risk innovations to solve some of humanity’s most complex challenges. Our biomedical research work focuses on a rare form of congenital heart defects called single ventricle heart disease. While this field is in its infancy, with limited knowledge of cause, risk, outcomes, or treatments, ...