AXLRx Novartis intelligence — Rare Disease in United States: 2 reports covering Rare Disease in United States.
Fabhalta covered via specialty pharmacy; complement franchise leverages the Soliris/Ultomiris access precedent.
The ATTR-CM vs ATTR-PN split, the wild-type/hereditary divide, and why Tc-PYP scintigraphy, not biopsy, is now the diagnostic gate for a largely undiagnosed population.
HAE pathophysiology, the Type I/II split, attack burden and the diagnostic-delay problem that defines the US in-market landscape.