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The authors emphasized the importance of early diagnosis and treatment with CGTs, especially for patients with genetic conditions. | Image credit: RFBSIP - stock.adobe.com

Cell and gene therapies can be life-changing for patients with certain conditions, but the process of receiving them poses barriers for patients and caregivers that require multistakeholder solutions, according to a white paper from the National Pharmaceutical Council.

Ifetroban may improve LVEF in patients with DMD. - REDPIXEL - stock.adobe.com

There is an unmet need for therapies targeting Duchenne muscular dystrophy (DMD)–related heart disease, and phase 2 results suggest ifetroban may improve left ventricular ejection fraction in patients with DMD.

MASH-related PLC is on the rise in young adult populations, causing concerns about how to best address the metabolic disease epidemic | image credit: https://gencraft.com/generate

As a growing number of young adults with primary liver cancer (PLC) related to metabolic dysfunction–associated steatohepatitis (MASH) age, developing coherent strategies to combat the metabolic disease epidemic is critical.