FDA/CDC

Firdapse approved: First treatment for rare autoimmune disorder


 

The Food and Drug Administration has approved amifampridine (Firdapse) as the first treatment for the rare autoimmune disorder known as Lambert-Eaton myasthenic syndrome, which causes the immune system to attack the neuromuscular junction and thereby disrupts the nerves’ ability to send signals to muscle cells. This causes fatigue and weakness in those affected, so they can experience difficulties with activities of daily living as a result.

FDA icon

The most common side effects included prickling sensation, upper respiratory tract infection, abdominal pain, and muscle spasms.

More information can be found in the FDA’s press announcement.

Recommended Reading

Spinraza shows motor improvements in older children with SMA type 1
MDedge Neurology
ATTR-ACT shows treatment breakthrough in amyloid cardiomyopathy
MDedge Neurology
Idiopathic inflammatory myopathies: Muscle biopsy no longer needed?
MDedge Neurology
Neurologic disease eventually affects half of women and one-third of men
MDedge Neurology
Planning for ventilator-dependent patients during natural disasters
MDedge Neurology
New Registry Offers Insight Into Opsoclonus-Myoclonus Syndrome
MDedge Neurology
Disability in Patients With Stiff Person Syndrome May Progress Faster Than Thought
MDedge Neurology
Does Thymectomy Benefit Patients With Anti-MuSK Myasthenia Gravis?
MDedge Neurology
Conference News Roundup—American Association of Neuromuscular & Electrodiagnostic Medicine
MDedge Neurology
CDC: No medical therapy can yet be recommended for acute flaccid myelitis
MDedge Neurology