Attend the 2022 Rett Syndrome National Summit
(Mountain View, Calif., April 14, 2022 PRNewswire=Yonhap) Adge Pharmaceuticals (“Adge”) is launching a new drug treatment for Rett Syndrome and Oste Dysplasia to support the US FDA’s regulatory requirements for expedited transport of lead assets to hospitals. We continue to develop our rare disease program.
Dr. Kalev Kask, CEO and Founder of Adge, will attend the ASCEND 2022 Rett Syndrome National Summit in Nashville, April 27-30, 2022, while preparing for the launch of the Rett Syndrome clinical program. .
As previously announced (see https://adgepharm.com/news/mar-22-2022/), Adge has secured a worldwide license for the small, clinical-stage oral molecule RO269228, originally developed by Roche for osteoporosis. . RO269228, also known as Elocalcitol, is a vitamin D analogue that has proven efficacy and safety in several phase 2 clinical trials. In addition, its mechanism of action is closely related to the resolution of the pathogenesis of several rare diseases, including Rett syndrome and osteogenesis imperfecta, which Adge has prioritized as an indication.
Rett syndrome is a neurodevelopmental disorder caused by mutations in the MECP2 gene, affecting almost exclusively girls. The syndrome is characterized by normal early growth and development, followed by developmental slowing, slowed growth of the brain and head, loss of ability to use the hand intentionally, characteristic hand movements, gait problems, seizures, autistic behavior, and intellectual disability. Rett syndrome is the second most common neurodevelopmental disorder in girls following Down syndrome, affecting approximately 1 in 9,000 to 10,000 women.
Osteogenesis imperfecta, also known as clavicle disease, is a group of genetic disorders that primarily affect the bones. People with this condition are prone to fractures, often with minor trauma or no apparent cause. Multiple fractures are common and can occur even before birth in severe cases. Osteogenesis imperfecta is a rare disease that affects regarding 1 in 10,000 to 20,000 people worldwide. About 25,000 to 50,000 people in the United States have this disease.
Adge Pharmaceuticals is a clinical-stage biopharmaceutical company focused on developing first-class therapies for rare indications.
출처: Adge Pharmaceuticals Inc.
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Source: PRNewswire Press Release
