Managing DMD Across the Lifespan
Another dimension of DMD care is providing resources and help to young people with DMD as they transition into adulthood. “In the past, we had limited treatment and mortality typically took place in the early 20s, so there weren’t a lot of patients who were adults. But as medication options have expanded and management of cardiac and respiratory failure has improved, we see a more significant proportion of adults who require adult-appropriate clinics — or, at the very least, specialists who are conversant in care or can provide care across the lifespan,” Dr. Brandsema said.
The DMD Care Considerations Working Group provides recommendations regarding care across the lifespan,9 as does the Adult North Star Network, of Muscular Dystrophy UK.17,18
Dr. Brandsema emphasized that, despite their disability, many adults with DMD “still engage with the community, and live life to its fullest.” It is to be hoped that, with ongoing research, earlier diagnosis, and improved treatment options, the future will look bright for people with DMD.
Dr. Brandsema has served as a consultant for Audentes, AveXis/Novartis, Biogen, Cytokinetics, Dyne, Edgewise, Fibrogen, Genentech, Marathon, Momenta/Janssen, NS Pharma, Pfizer, PTC Therapeutics, Sarepta, Scholar Rock, Takeda, and WaVe. He is on the medical advisory council member for Cure SMA and is a site investigator for clinical trials with Alexion, Astellas, AveXis/Novartis, Biogen, Biohaven, Catabasis, CSL Behring, Cytokinetics, Dyne, Fibrogen, Genentech, Ionis, Lilly, ML Bio, Pfizer, PTC Therapeutics, Sarepta, Scholar Rock, Summit, and WaVe. Ms. Kaschak has nothing to disclose.
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