Celebrating the Most Expensive Drug for the Disease You Never Heard Of & More — This Week in RARE Daily

March 29, 2024

This Week in RARE Daily is a feature from Global Genes where you can get a quick rundown of the top 5 headlines in the rare disease space from our editorial staff. Here are top stories from this past week, March 22-28, 2024:

Celebrating the Most Expensive Drug for the Disease You Never Heard Of
Lenmeldy, the first gene therapy for metachromatic leukodystrophy, won approval from the FDA last week, but price and access are major concerns now.

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Researchers Find Genetic Switch Underlying Rare Inflammatory Diseases
Researchers have discovered how the body’s inflammation machinery can get stuck and cause rare genetic inflammatory diseases.

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FDA Approves Merck’s Winrevair for Adults with Pulmonary Arterial Hypertension
Winrevair is the first FDA-approved activin signaling inhibitor therapy for PAH, representing a new class of therapy that works by improving the balance between pro- and anti-proliferative signaling.

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Researchers Unlock Genetics of Ultra-Rare Neurodevelopmental Disorder
Researchers at Ottawa’s Faculty of Medicine have revealed how gene mutations work to cause Borjeson-Forssman-Lehmann Syndrome, an ultra-rare, X-linked neurodevelopmental disorder

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Stoke Surges After Reporting Positive Results from Dravet Syndrome Study
Stokes Therapeutics reported positive new data from studies for those aged 2 to 18 with Dravet syndrome who were treated with STK-001.

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