Exciting breakthroughs in rare disease treatments mean more patients are gaining access to cutting-edge therapies - and specialty pharmacies are playing a crucial role in delivering these medications with expert support. But what exactly do they do, and when do they come into play?
AllMyHealth’s latest guide breaks it all down - exploring how specialty pharmacies work, their expanding role in patient care, and how individuals can engage with them. Plus, we spotlight the specialty pharmacy Biologics by McKesson which is the sole distributor of Skyclarys treatment.
Available in both text and audio formats - dive in today and share with your community!
In a novel therapeutic approach, Dong et al. (2025) identified a peptide that disrupts a specific protein interaction, leading to increased frataxin levels in Friedreich's ataxia (FRDA) patient cells. By targeting this interaction, the study demonstrated notable improvements in mitochondrial function, highlighting the potential of peptide-based therapies for FRDA.
This research suggests a new therapeutic avenue by modulating protein interactions to restore cellular balance in FRDA. The findings emphasize the importance of targeting disease-specific molecular pathways, opening possibilities for further drug development aimed at improving mitochondrial health in FRDA patients.
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