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As anyone affected by a rare disease knows, treating the illness while trying to go about everyday life is an expensive undertaking.  But exactly how expensive — in terms of direct and indirect costs across rare disease populations — might still come as a surprise: almost…

Editor’s note: This story was updated March 31, 2021, to clarify that Firdapse is currently the only therapy approved in the U.S. to treat adults with LEMS. Ruzurgi also is approved in the U.S. to treat children and adolescents, ages 6–16, with LEMS. Development of a new long-acting formulation of Firdapse…

In the U.S., treatments for rare diseases such as Lambert-Eaton myasthenic syndrome (LEMS) account for only 11% of total medical invoice spending in the country, according to a recent report. The report, “Orphan Drugs in the United States: Rare Disease Innovation and Cost Trends Through 2019,” is…

Many diseases have their own awareness color — breast cancer is pink, muscular dystrophy is green, and AIDS is red, for example — but what’s the significance of pink, green, blue, and purple lights side-by-side? These are the colors most often used to represent Rare Disease Day. This…

For the more than 25 million Americans living with rare diseases, including Lambert-Eaton myasthenic syndrome (LEMS), medical emergencies can pose unique challenges: because these disorders are by definition rare, first responders or emergency personnel may not be aware of a person’s condition, or familiar with the appropriate treatment.

Diagnosed with sickle cell disease as a 6-month-old, Tristan Lee has faced a lot of challenges over his 37 years of life. But from a young age, he also learned how to turn those trials into triumphs. At age 9, a stroke due to his disease left him paralyzed…

Firdapse (amifampridine) developer Catalyst Pharmaceuticals is supporting Rare Disease Day 2021 by sponsoring and participating in a virtual Facebook Live event on Friday, Feb. 26, at 4 p.m. ET. The live event, streaming on Facebook, will feature a discussion panel that includes a patient advocate with Lambert-Eaton…

Rare Disease Day at NIH, organized by the National Institutes of Health (NIH) and taking place on March 1, will feature panel discussions, patient stories, research updates, TED-style talks, and a presentation by a Nobel laureate recently recognized for her work on a gene editing tool. The free, virtual…

People with rare disorders have a worse healthcare experience than those affected by chronic diseases, according to the results of an international survey conducted by Eurordis-Rare Diseases Europe. Indeed, rare disease patients overall give their healthcare experience a medium-low rating, of 2.5 on a scale of 1 to 5,…

Scores of virtual events are afoot around the world to mark Rare Disease Day 2021 on Feb. 28. The activities are focused on heightening awareness about rare diseases and the hundreds of millions of individuals they are thought to affect. Patients, caregivers, and advocates worldwide will sport denim ribbons…