Latest News


Rare disease spotlight


We are so excited to feature an incredible lineup of community artists for PPMD’s Community Concert: Harmony Together tomorrow evening, November 22 at 7 PM EST! Meet our featured community artists:

AVROBIO is now enrolling males with classic #Fabry disease for a Phase 1/2 clinical trial to study AVR-RD-01. To learn more and find out if you’re eligible, contact 1-877-330-5216 and visit

Novartis is conducting a clinical trial in patients with sickle cell disease 16 years and older with chronic kidney disease. The Phase II STEADFAST clinical trial is now enrolling.

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