Rare Diseases Constitute a ‘Public Health Issue,’ NCATS Director Warns
With 250 rare diseases newly identified every year, scientists can barely keep up — even as the healthcare system fails millions of Americans whose ... Read more
Updates
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Ruzurgi Approved in United States as First Treatment for Children with LEMS
The U.S. Food and Drug Administration (FDA) has approved Ruzurgi (amifampridine) for the treatment of children ages 6–17 with Lambert-Eaton myasthenic syndrome (LEMS). The ... Read more -
SCLC Patients with LEMS Have Better Long-term Survival, Study Suggests
Patients who have Lambert-Eaton myasthenic syndrome (LEMS) associated with small cell lung cancer (SCLC) seem to have better long-term survival than cancer patients without ... Read more -
LEMS Prevalence in U.S. Similar to Europe, Large U.S. Study Reports
Lambert-Eaton myasthenic syndrome (LEMS) has a similar prevalence and incidence in the U.S. compared with European populations, and most patients get better with therapy, ... Read more -
Paraneoplastic Cerebellar Degeneration and LEMS Linked to Rare Throat Tumor, Case Report Shows
A case of paraneoplastic cerebellar degeneration (PCD) and Lambert-Eaton myasthenic syndrome (LEMS) was associated with a hormone-related tumor in a patient’s oropharynx, or the ... Read more
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Screening Leads to Simultaneous Lambert–Eaton Myasthenic Syndrome and Limbic Encephalitis Diagnoses, in Unusual Case Report
A recent case report of a man diagnosed with simultaneous Lambert-Eaton myasthenic syndrome (LEMS) ... -
Mouse Model of LEMS Provides New Tool to Develop Therapies
An animal model of Lambert-Eaton myasthenic syndrome (LEMS) has enabled better understanding of what ... -
Brexit Could Have Real Effects for UK Rare Disease Patients, Experts Warn
Madeline Collin, a 24-year-old activist with Gaucher disease, worries that patients like her will ... -
Moral Dilemmas Complicate Treatment of Rare Diseases, Says Israeli Bioethicist
With each new advance in medicine comes ethical dilemmas, from fertility treatments and newborn ... -
NIH Rare Disease Day Highlights Joint Networks Advancing Array of Research
Rare diseases affect about 30 million Americans — roughly the same number as those ... -
Firdapse Found to be Effective for Lambert-Eaton Myasthenic Syndrome, Phase 3 Trial Shows
Firdapse led to clinically significant improvements, compared with a placebo, in adult patients with Lambert-Eaton ...
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FDA Accepts Catalyst’s NDA for Firdapse with Priority Review Status for LEMS
News
The U.S. Food and Drug Administration has accepted Catalyst Pharmaceuticals’ new drug application (NDA) for Firdapse (amifampridine phosphate) for Lambert-Eaton myasthenic syndrome (LEMS), with priority review status. Firdapse is a formulation of amifampridine phosphate in development for the treatment of LEMS as ... Read more