Lambert Eaton Myasthenic Syndrome News and Research

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A bitter battle over the ‘orphan drug’ program leaves patients’ pocketbooks at risk

A prescription drug that helps Lore Wilkinson walk and talk despite a rare muscle disease cost her so little for more than a decade that she didn't even use her insurance to pay for it.

22 Feb 2023

FDA approves new drug for treatment of rare autoimmune disorder

The U.S. Food and Drug Administration today approved Firdapse (amifampridine) tablets for the treatment of Lambert-Eaton myasthenic syndrome (LEMS) in adults.

29 Nov 2018

Intravenous immune globulin effective treatment for GBS, CIDP

Intravenous immune globulin (IVIg) is an effective treatment for certain disorders of the nerve and muscles, including Guillain-Barré syndrome (GBS) and a form of neuropathy called chronic inflammatory demyelinating polyneuropathy (CIDP), according to a guideline issued by the American Academy of Neurology.

27 Mar 2012

BioMarin reports $0.5 million GAAP net loss for second-quarter 2010

BioMarin Pharmaceutical Inc. today announced financial results for the second quarter of 2010. GAAP net loss was $0.5 million ($0.01 per diluted share) for the second quarter of 2010, compared to GAAP net income of $1.3 million ($0.01 per diluted share) for the second quarter of 2009. Non-GAAP net income was $8.6 million ($0.08 per diluted share) for the second quarter of 2010, compared to non-GAAP net income of $9.0 million ($0.09 per diluted share) for the second quarter of 2009.

3 Aug 2010

BioMarin Pharmaceutical introduces Firdapse for LEMS in the E.U.

BioMarin Pharmaceutical Inc. announced today that Firdapse™ (3,4-diaminopyridine) is now commercially available in the European Union (E.U.) for the treatment of the rare autoimmune disease Lambert Eaton Myasthenic Syndrome (LEMS). Launching immediately in Germany and the UK, the company expects to subsequently launch Firdapse in all major European markets by the end of 2010.

19 Apr 2010

BioMarin Pharmaceutical receives marketing approval for amifampridine phosphate

BioMarin Pharmaceutical Inc. announced today that the European Commission has granted marketing approval for 3,4-diaminopyridine (amifampridine phosphate), for the rare autoimmune disease Lambert Eaton Myasthenic Syndrome (LEMS).

6 Jan 2010

BioMarin Pharmaceutical receives FDA orphan drug designation for 3,4-DAP

BioMarin Pharmaceutical Inc. announced today that the Food and Drug Administration (FDA) has granted orphan drug designation for 3,4-diaminopyridine (3,4-DAP), amifampridine phosphate, for the rare autoimmune disease Lambert Eaton Myasthenic Syndrome (LEMS). 3,4-DAP has previously received orphan drug designation in the E.U.

30 Nov 2009

BioMarin Pharmaceutical announces the acquisition of Huxley Pharmaceuticals

BioMarin Pharmaceutical Inc. announced today that it has acquired Huxley Pharmaceuticals, Inc. (Huxley), which has rights to a proprietary form of 3,4-diaminopyridine (3,4-DAP), amifampridine phosphate, for the rare autoimmune disease Lambert Eaton Myasthenic Syndrome (LEMS).

27 Oct 2009