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Health officials identify three more cases of mysterious brain disease in New Brunswick theglobeandmail.com - get the latest breaking news, showbiz & celebrity photos, sport news & rumours, viral videos and top stories from theglobeandmail.com Daily Mail and Mail on Sunday newspapers.
F. Hoffmann-La Roche Ltd: Roche receives positive CHMP opinion for Evrysdi, the first and only at home spinal muscular atrophy (SMA) treatment with proven efficacy in adults, children and infants two months and older More than 2,500 patients now treated with Evrysdi in clinical trial, compassionate use and real-world settings Evrysdi is approved in seven countries, submitted in 30 more Basel, 26 February 2021 - Roche (SIX: RO, ROG; OTCQX: RHHBY) today announced that the European Medicines Agency (EMA) Committee for Medicinal Products for Human Use (CHMP) has recommended the approval of Evrysdi (risdiplam) for the treatment of 5q spinal muscular atrophy (SMA) in patients 2 months of age and older, with a clinical diagnosis of SMA Type 1, Type 2 or Type 3 or with one to four SMN2 copies. SMA is a leading genetic cause of death in infants and 5q SMA is the most common form of the disease. SMA causes progressive muscle weakness and atrophy, and significant unmet need rema ....
FIREFISH Part 1 data show treatment with Evrysdi at 12 months helped 90% of these infants survive without permanent ventilation and 33% sit without support, a key motor milestone not normally seen in the ....
Hirayama Disease What is Hirayama Disease? Hirayama disease, also known as monomelic amyotrophy, Sobue disease or Juvenile non-progressive amyotrophy is a rare and incurable disease that was first described in 1959 by Dr. Keizo Hirayama, a neurologist, attached to the Chiba University School of Medicine in Japan. It mainly affects young males aged between 15-25 years, generally after they experience their adolescent growth spurt. Technically, it is termed as a lower motor neuron disorder. Classical findings include muscle wasting (atrophy ) and weakness of the forearms and hands, either on one or both sides. There is, however, no change in sensation and the patient doesnt experience any pain. There may be involuntary muscle twitching (fasciculations), but these are rare. The disease usually progresses for 2-5 years before stabilizing or plateauing. After plateauing, improvement in the symptoms does not occur. However, it does not worsen either. ....
DomoSafety, a digital health leader offering a private cloud platform for patient monitoring and decision support for home care, has launched a pilot project with Roche Pharma (Switzerland) Ltd. The project aims at improving the quality of life of people living with Spinal Muscular Atrophy, a rare neuromuscular disease. Spinal muscular atrophy (SMA) is a severe, inherited, progressive neuromuscular disease that causes devastating muscle atrophy and disease-related complications. It is the most common genetic cause of infant mortality and one of the most common rare diseases, affecting approximately one in 11,000 babies. SMA leads to the progressive loss of nerve cells in the spinal cord that controls muscle movement. Depending on the type of SMA, an individual’s physical strength and their ability to walk, eat or breathe can be significantly diminished or lost. ....