Medical Education Library

Rare Neurological Disease Special Report


 

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Our sixth annual Rare Neurological Disease Special Report is our biggest issue yet. It is very gratifying to know we are part of the rare disease community and witness to some of the exciting developments that are transforming this field. There are many newly approved therapies highlighted in the articles in this issue, as well as brief profiles of a number of research groups within the NIH’s Rare Diseases Clinical Research Network and an intriguing profile of how the Netflix show Diagnosis used crowdsourcing to solve medical mysteries, many of which involved rare neurologic conditions. That’s just a sampling of what this issue has to offer. There are too many articles to mention each one, but I hope you take the time to read the entire issue.

Glenn S. Williams, vice president, group editor, Neurology Reviews

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Recommended Reading

FDA approves Givlaari for treatment of acute hepatic porphyria
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Study delineates spectrum of Dravet syndrome phenotypes
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Spotlight on SMA: The urgent need for early diagnosis in spinal muscular atrophy
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Expedited review programs not shortening drug development
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Zilucoplan improved efficacy outcomes in myasthenia gravis
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Eculizumab reduces relapse-related hospitalizations in patients with NMOSD
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Inebilizumab benefits patients with NMOSD
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Upcoming vaccine may offset surge in polio subtypes
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