Written by Mike Dobbyn “Life expectancy: Early to late teens.” It was March 20th, 2019, and I was reading my son’s obituary on my iPhone while sitting in the geneticist’s…
You might have heard of Orphan Drug designation - and maybe even Rare Pediatric Disease designation. But have you heard of Regenerative Medicine Advanced Therapy (RMAT) designation? Described in Section…
Up to 10% of heart failure cases could be caused by cardiac amyloidosis. Yet amyloidosis remains underdiagnosed and misunderstood. Broadening our understanding of this rare disease could contribute to earlier…
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