Patient Engagement & Rare Diseases Podcast

FDA approves Ultragenyx's Fayuvi as the first treatment for Sanfilippo syndrome type A, Lisata Therapeutics acquires Marea Therapeutics as mAbxience partners with Sandoz on a hemophilia A biosimilar, and Joe Cunningham argues that executive drug-pricing deals alone won't fix costs.

Lisata Therapeutics acquires Marea Therapeutics and secures $225 million in concurrent financing, while mAbxience and Sandoz partner to develop and globally commercialize an emicizumab biosimilar.

FDA approved Ultragenyx's Fayuvi (rebisufligene etisparvovec-hopf) as the first treatment for MPS IIIA Sanfilippo syndrome type A.

Former Rep. Joe Cunningham (D-SC), now spokesperson for the Pharmaceutical Reform Alliance, argues that codifying Most-Favored-Nation pricing into law is the real fix for drug costs.

Former U.S. Representative Joe Cunningham (D-SC), spokesperson, Pharmaceutical Reform Alliance. dismisses the pharmaceutical industry's innovation argument against MFN pricing.

Inovalon's Jen Lamppa on why inaccurate self-reported data is clinical trial enrollment's costliest problem — and how RWD fixes it.

Five major pharma stories in one day — Novo's rebrand and AI bets, Bayer's 30-year CKD first, GSK's T-cell engager, and Sanofi's portfolio exit.

Inizio's Elaine Ferguson on why enabling purpose-driven employees to make a meaningful difference is as important as any formal talent strategy.