International Fibrodysplasia Ossificans Progressiva Awareness Day is on April 23: Spreading Rare Disease Awareness
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International Fibrodysplasia Ossificans Progressiva Awareness Day is on April 23: Spreading Rare Disease Awareness

International Fibrodysplasia Ossificans Progressiva (FOP) Awareness Day is recognized each year on April 23rd. This is a time for spreading awareness about this rare disorder among both the general public…

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Update: Phase 3 Data Published on Ganaxalone for CDD
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Update: Phase 3 Data Published on Ganaxalone for CDD

According to a recent article in Businesswire, The Lancet has published data from the third phase of the Marigold trial. This clinical trial evaluated ganaxalone - marketed as ZTALMY - as a…

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Can Music Therapy Relieve SCD Pain?
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Can Music Therapy Relieve SCD Pain?

Many patients with sickle cell disease (SCD) experience “pain crises,” or periodic episodes of extreme pain. These pain crises occur when sickle-shaped red blood cells block the flow of blood…

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Donidalorsen Reduces HAE Attacks, Study Shows
Hand swollen during hereditary angioedema attack. Sourced from Wikimedia Commons

Donidalorsen Reduces HAE Attacks, Study Shows

In patients with hereditary angioedema (HAE), the C1-inhibitor blood protein does not function properly, allowing bradykinin peptides to build up in the body. These then cause inflammation and fluid leakage;…

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