The CheckRare Brief

The CheckRare Brief

A weekly roundup of the latest news in rare disease. The CheckRare Brief delivers a concise roundup of the week's most important developments in rare diseases. Each episode covers FDA approvals, clinical trial updates, conference highlights, scientific publications, and industry news that matter to healthcare professionals, researchers, advocates, and the rare disease community. Produced by CheckRare, The CheckRare Brief helps you stay informed—in just a few minutes each week.

Episodes

August 12, 2026 7 mins
On this week’s episode of The CheckRare Brief, we discuss FDA’s approval of Orzeyful (oveporexton) to treat patients with narcolepsy type 1, CAMP4’s first-in-human clinical trial for patients with SYNGAP1-related disorders, and BioMarin’s termination for their ENPP1 deficiency program following mixed results from their phase 3 clinical trial.

The US Food and Drug Administration (FDA) approved Takeda&r...
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The CheckRare Brief delivers a concise summary of the week's most important developments in rare diseases. Each episode covers FDA approvals, clinical trial updates, conference highlights, scientific publications, and industry news that matter to healthcare professionals, researchers, advocates, and the rare disease community.

Madaline Spencer, Podcast Producer; James Radke, PhD; Education Director; Peter Ciszewski,  Fo...
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