Sep 19, 2024 · Spinal muscular atrophy (SMA) is characterized by muscle weakness and atrophy resulting from progressive degeneration and irreversible loss of the anterior horn ...
Missing: orphan | Show results with:orphan
Sep 17, 2024 · Spinocerebellar ataxia (SCA) is a progressive, degenerative, genetic disease with multiple types, each of which could be considered a neurological condition ...
Sep 23, 2024 · Although the definition of a rare disease (sometimes called an “orphan disease”) varies in different countries, an individual disease is typically defined ...
5 days ago · FDA's Orphan Products Grants Program awards grants to clinical investigators to support the development of safe and effective medical products for patients ...
Sep 9, 2024 · Treatments for rare diseases are called orphan drugs, and their exact definition varies across countries. ... Atrophy (SMA), a rare genetic disorder. AI ...
Sep 19, 2024 · Poland Syndrome is a condition characterized by the absence or underdevelopment of the pectoralis major muscle and other malformations on.
Sep 26, 2024 · Orphan condition: 5q spinal muscular atrophy. Authorised orphan indication: Spinraza is indicated for the treatment of 5q Spinal Muscular Atrophy. Orphan ...
Sep 10, 2024 · Now that we have a definition of an orphan device in the EU, research activities can be targeted at this specific sub-type of medical devices.
Sep 13, 2024 · ALF occurs when the liver suddenly loses its ability to function in a person without a pre-existing liver disorder, and carries a high morbidity and mortality ...
Sep 11, 2024 · Freeman-Sheldon syndrome (FSS) or “whistling face syndrome” is an exceptionally rare disorder present before birth (congenital) that primarily affects muscles ...
Missing: atrophy | Show results with:atrophy
