How can the safety and efficacy of gene therapies be rigorously evaluated?

Muscle loss, or muscle atrophy, is a loss of muscle mass. It becomes visible over time. Muscle wasting causes limitations in everyday movements and can have ... Cure SMA provides support to patients and families affected by spinal muscular atrophy and funds and directs research leading the way to a cure for SMA. Nov 17, 2022 ... A mild degree of brain atrophy is not always a concern. Substantial brain atrophy can be associated with major neurological diseases, such as a ... What is geographic atrophy? Geographic atrophy (GA) is the scientific term for late stage dry aged-related macular degeneration (AMD). It is called geographic ... It's sometimes called Werdnig-Hoffmann disease or infantile-onset SMA. Children with type 1 have limited movement, can't sit without support, and have trouble ... Dec 31, 2023 ... There are three types of muscle atrophy: physiologic, pathologic, and neurogenic. Physiologic atrophy is caused by not using the muscles enough. Jul 4, 2022 ... Signs and symptoms of vaginal atrophy · Bleeding and discomfort during and after intercourse. · Frequent urination and/or urine leakage. Sep 24, 2024 ... The term metaplastic (chronic) atrophic gastritis, also referred to as gastric atrophy, is used to describe a form of chronic gastritis that ... In GA, areas of the central macula experience cell death (atrophy). These areas can grow and may result in a dim or blind spot in your vision. GA often first ... Multiple system atrophy (MSA), formerly called Shy-Drager syndrome, is a rare condition of the brain and nervous system. MSA affects the body's ability to ...