FDA CDER’s Accelerating Rare Disease Cures Program

Revumenib + Chemotherapy in Newly Diagnosed AML With NPM1 Mutation: Phase 3 REVEAL-ND Study by CheckRare Staff| Published on: Sep 23, 2026

My name is Dr. Joshua Zeidner, and I’m a Professor of Medicine and Chief of Leukemia Research at the University of North Carolina Lineberger Cancer Center. I’m here today to raise awareness around an active global, phase 3, double-blind trial evaluating a combination regimen for participants with a specific type of acute myeloid leukemia or AML. https://checkrare.com/revumenib-chemotherapy-in-newly-diagnosed-aml-with-npm1-mutation-phase-3-reveal-nd-study/

FDA Approves New Therapies for MPS IIIA and SMA | The CheckRare Brief Ep 8 by CheckRare Staff| Published on: Sep 23, 2026

This week we discuss the FDA’s approval of Isembyld for SMA, the approval of a gene therapy for MPS IIIA, and the FDA’s Center for Devices and Radiological Health new Rare Disease Impact Initiative. https://checkrare.com/fda-approves-new-therapies-for-mps-iiia-and-sma-the-checkrare-brief-ep-8/

New Rare Disease Center of Excellence and FDA Advances in Rare Disease | The CheckRare Brief Ep 9 by CheckRare Staff| Published on: Sep 29, 2026

This week on The CheckRare Brief, we are joined by Eugene Lee, CMI Media Group’s Group President, Specialty Communications, for an industry perspective on some of the biggest developments in rare disease. https://checkrare.com/new-rare-disease-center-of-excellence-and-fda-advances-in-rare-disease/

MOST READ IN Cancer Science & Oncogenesis (+...)

https://read.qxmd.com/collection/1133?ecd=wnl_readmost_261002

Novo bets big on Hengrui’s weight loss pill in $2.6B deal Pharma deals, regulatory updates, new treatments and more led the news this week. Brought to you byDDN editorial team

Novo bets big on Hengrui’s weight loss pill in $2.6B deal Pharma deals, regulatory updates, new treatments and more led the news this week. Brought to you byDDN editorial team https://www.drugdiscoverynews.com/novo-bets-big-on-hengrui-s-weight-loss-pill-in-2-6b-deal-17563 In an increasingly competitive oral GLP-1 market, Novo set its sights on Hengrui Pharma’s GLP-1/GIP dual receptor agonist that boasts the potential for once-weekly oral dosing. The company agreed to pay Hengrui $300 million up front for rights to the drug, HRS-1596, with milestone payments up to $2.6 billion. Currently, the drug is in Phase 1 trials in China to treat patients with type 2 diabetes and weight management. “Novo has pioneered the field of oral peptides and as leaders, we continuously look to advance our broad and deep oral pipeline across obesity, diabetes and other cardiometabolic diseases through our strong internal capabilities and leading external innovation," said Martin Holst Lange, Executive Vice president, Research & Development and Chief Scientific Officer at Novo in the statement. – Allison Whitten

Raising the profile of NF1 and advocating for medical curiosity (+...)

https://rarerevolutionmagazine.com/digital_spotlights/nf1/ We are delighted to launch our newest RARE in Focus all about neurofibromatosis type 1(NF1). Huge thank you to our sponsors Childhood Tumour Trust who celebrate their 10th anniversary as a charity in 2026. Dive in to hear from Kirsten Samuel, Sheelagh Harwell, Ellie Day and Rakie Ayola. https://rarerevolutionmagazine.com/digitalspotlight/raising-the-profile-of-nf1-and-advocating-for-medical-curiosity/