What are the regulatory pathways for approving new therapies for rare diseases?

Summary · Spinal muscular atrophy is an inherited condition. · The nerve cells that service the muscles don't work properly, causing muscle weakness and wasting ... 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 ... Jan 12, 2021 ... Excessive muscle loss is a bad prognostic index of a variety of diseases ranging from cancer, organ failure, infections and unhealthy ageing. Dec 31, 2023 ... There are three types of muscle atrophy: physiologic, pathologic, and neurogenic. Physiologic atrophy is caused by not using the muscles enough. Dry (atrophic) AMD is also known as geographic atrophy. It occurs when retinal cells gradually die (atrophy), leading to patches of missing retina and loss of ... Jul 18, 2023 ... What is Atrophy? Atrophy is a type of tissue wasting that occurs as a result of a disease process that is outside of the tissue that is wasting. Progressive retinal atrophy (PRA) is a group of degenerative diseases that affect photoreceptor cells. With this disease, the cells deteriorate over time, ... vaginal atrophy ... A condition in which the tissues lining the inside of the vagina (birth canal) become thin, dry, and inflamed. This is caused by a decrease in ... Background:The presentation of symptoms for multiple system atrophy (MSA) varies. Because there are no specific markers for its clinical diagnosis, the. NCI's Dictionary of Cancer Terms provides easy-to-understand definitions for words and phrases related to cancer and medicine.