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Primary Periodic Paralysis 101
 ibreakstock/Shutterstock

Marked by muscle weakness that begins early in life, periodic paralysis renders patients immobile in episodes that last anywhere from hours to days.

Rare Diseases

Satralizumab BLA Accepted by FDA for Treatment of NMOSD

November 13, 2019
  • Marco Meglio

Study data demonstrates that treatment with satralizumab reduces risk of relapses in patients with neuromyelitis optica spectrum disorder.

  • READ >>
FDA

FDA Rejects Duchenne Muscular Dystrophy Drug Golodirsen, Citing Safety Concerns

August 23, 2019
  • Jenna Payesko

The regulatory agency cited 2 concerns in the complete response letter: the risk of infections related to intravenous infusion ports and renal toxicity.

  • READ >>

Developing Targeted Therapeutics in Angelman Syndrome

July 16, 2019
  • Alex Kolevzon, MD

The genomic revolution has led to increasing opportunities to address complex neurodevelopmental conditions, taking a genetics-first approach.

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Genetics DNA genetic testing

Genetic Testing for Huntington Disease

July 3, 2019
  • Amir Bishay, MD

The profound consequences of the results guide decisions about testing. Who should be tested? And, just as important, who should not?

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Angelman syndrome researcher

Phase 2 Trial for Angelman Syndrome Underway

June 25, 2019
  • Heidi Moawad, MD
  • Cesar Ochoa-Lubinoff, MD, MPH, FAAP

In this podcast, Heidi Moawad, MD interviews Cesar Ochoa-Lubinoff, MD, MPH, FAAP, about recent encouraging clinical trial results in patients with Angelman syndrome.

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7 New Drugs for Neurologists

April 5, 2019
  • Julie Bowen

Here’s a brief look at therapies for neurological disorders that the FDA has approved within the past 6 months.

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Teenager Who Sleeps Up to 20 Hours a Day

March 15, 2019
  • Heidi Moawad, MD

EEG and brain MRI findings are normal—what do you suspect?

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Neuropsychiatric Symptoms in Huntington Disease: A 5-Question Quiz

February 28, 2019
  • Julie Bowen

Are you up-to-date on recent guidelines for the management of agitation, anxiety, apathy, psychosis, and sleep disorders in patients with Huntington disease? Take this brief quiz to find out.

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New Guidelines on Management of Neuropsychiatric Symptoms in Huntington Disease

February 14, 2019
  • Veronica Hackethal, MD

Some good news about Huntington disease: most of the behavioral symptoms can be managed with treatments developed for other conditions.

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5 Qs on Vaccines and Guillain-Barre Syndrome

December 13, 2018
  • Veronica Hackethal, MD

What is the most common risk factor for Guillain-Barré Syndrome? Plus 4 other quick questions to test your knowledge. 

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