**Hope for Rare Disease Patients as Medicines Watchdog Approves Groundbreaking Treatments** In a significant breakthrough for families affected by a devastating rare disease, the National Institute for Health and Care Excellence (Nice) has given the green light to two pioneering treatments for spinal muscular atrophy (SMA). SMA is a rare and progressive muscle-wasting condition that affects hundreds of children in the UK, causing muscle weakness and respiratory problems. The approval of these life-changing medications is being hailed as a "lifeline" by parents, who have been fighting for access to these treatments for years. With these treatments now available, patients who would benefit from them will be able to receive one of the two approved drugs, offering new hope for improved survival rates and a better quality of life for those affected by this debilitating disease.
Medicines watchdog approves two treatments for patients with spinal muscular atrophyHundreds of children with a rare muscle-wasting disease will be able to receive two drugs that can improve their survival in a move parents hailed as a “lifeline”.The National Institute for Health and Care Excellence (Nice) has published final draft guidance recommending that any patient who would benefit can have either drug. Continue reading...
