Tag : rare diseases

Neurology

FDA OKs First Treatment for Lambert-Eaton Myasthenic Syndrome

Newsemia
Amifampridine (Firdapse) is approved for adults with Lambert-Eaton myasthenic syndrome, a rare autoimmune disorder characterized by muscle weakness. FDA Approvals Source link...
Neurology

EMA Panel Backs First Oral-Only Drug for Deadly Disease

Newsemia
Fexinidazole is for the treatment of human African trypanosomiasis caused by Trypanosoma brucei gambiense in adults and children aged 6 years and older. International Approvals...
Neurology

Periodic Paralysis: Shedding Light on a Rare Neurologic Disorder

Newsemia
This infrequent neuromuscular disorder is commonly mistaken for a multitude of other disorders, but diagnosis can be confirmed with key clues and genetic testing. Medscape...
Neurology

ATTR-ACT: Tafamidis Reduces Mortality in Amyloid Cardiomyopathy

Newsemia
A drug that binds transthyretin reduced all-cause mortality and cardiovascular hospitalizations and slowed decline in functional capacity in patients with transthyretin amyloid cardiomyopathy. Medscape Medical...
Neurology

FDA OKs Patisiran (Onpattro) for Polyneuropathy in hAATR

Newsemia
Patisiran is the first FDA-approved treatment for polyneuropathy caused by hATTR and the first RNA-based therapy to treat a rare disease. FDA Approvals Source link...

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