Rare Diseases Update by LucidQuest Episode 9

🔬 Rare Disease Breakthroughs
Welcome to this week’s edition of Rare Disease Updates by LucidQuest — your trusted source for the latest clinical milestones and regulatory approvals in rare disease innovation.

In this episode, we cover major developments across hematology, immunology, nephrology, and genetic disorders:

✅ The FDA grants Orphan Drug Designation to rilzabrutinib for wAIHA and IgG4-RD, two conditions with no approved therapies.
✅ UPLIZNA becomes the first and only FDA-approved treatment for adults with IgG4-RD, reducing disease flare risk by 87 percent.
✅ BioMarin's PALYNZIQ shows significant efficacy in adolescents with phenylketonuria (PKU) in the Phase 3 PEGASUS trial.
✅ ResVita Bio's RVB-003 earns Orphan Drug status for Netherton Syndrome, following Rare Pediatric Disease Designation.
✅ Novartis' Vanrafia (atrasentan) receives FDA accelerated approval for IgA nephropathy, reducing proteinuria by 36.1 percent.
✅ Sarepta's ELEVIDYS gene therapy trials resume in the EU after an independent safety review supports its benefit-risk profile.
✅ Rallybio discontinues RLYB212 for FNAIT, shifts focus to next-gen complement therapies.
✅ Opus Genetics shares promising pediatric data for retinal gene therapy OPGx-LCA5, improving vision after just one dose.

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Видео Rare Diseases Update by LucidQuest Episode 9 канала LucidQuest