Boy practicing golf on a putting green, holding a putter with a large yellow “f(x)” graphic overlaid; small text in the corner reads “Tripp, Age 6— Living with Duchenne.”

Tripp, Age 6
Living with Duchenne 

Scientific illustration of a dystrophin protein connecting muscle cell structures at the membrane, highlighting its role in maintaining muscle integrity.

Functional dystrophin and the science of muscle integrity

Learn more about critical domains that make up the dystrophin protein and explore the key components that make it essential for muscle integrity.

DIVE DEEPER INTO DYSTROPHIN

Boy wearing glasses and a light-colored fedora hat sitting on a couch indoors, drinking from a green coconut through a straw. Text reads in the bottom right corner "Alex, Age 19 — Living with Duchenne."

Alex, Age 19
Living with Duchenne

The future of Duchenne: What's next?

Hear from Dr Finanger, Caeden, and Lianette as they share what matters most moving forward.

WATCH THEIR PERSPECTIVES

Blue graphic displaying the Functional Improvement Equation including “Quantity,” “Quality,” and “Distribution” with highlighted initials Q, Q, and D, plus symbols beside each term, and “Functional improvement” written below a line.

We need to understand dystrophin in a much more nuanced way. We really need to understand all 3 of these aspects. And then importantly, how those 3 aspects translate to benefit for a patient.

Erika Finanger, MD, MS
Pediatric Neurologist and Neuromuscular Specialist

People living with Duchenne want more function7

Bracket
71 animation

of ambulatory caregiver-patient pairs (n=20) reported that they would be satisfied with a treatment that maintained their current level of functioning or stabilized the disease

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of non-ambulatory caregiver-patient pairs (n=13) reported that they would want to see a treatment improve independence in activities of daily living, acknowledging that this desire might not be realistic

Based on a 2023 qualitative study published in the Journal of Patient-Reported Outcomes examining patient and caregiver perspectives on the impact of Duchenne, including expectations for future treatments. The study was conducted in a manner that involved interviewing 46 caregiver-patient pairs. Participants included males aged ≥4 years with genetically confirmed Duchenne (ambulatory, n=28; non-ambulatory, n=18) and adult caregivers aged ≥18 years (n=42).

Close-up of a young man’s hand holding a black marker and writing on a white surface, with part of the side of the head visible in the foreground. A large red “f(x)” graphic is overlaid. Small text reads in the bottom left "Caden, Age 19 Living with Duchenne"
Close-up of a young man’s hand holding a black marker and writing on a white surface, with part of the side of the head visible in the foreground. A large red “f(x)” graphic is overlaid. Small text reads in the bottom left "Caden, Age 19 Living with Duchenne"

Caeden, Age 19
Living with Duchenne

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REFERENCES:

1. Bengtsson NE, Tasfaout H, Chamberlain JS. Mol Ther. 2025;33(5):2035-2051. 2. Bez Batti Angulski A, Hosny N, Cohen H, et al. Front Physiol. 2023;14:1183101. 3. Elangkovan N, Dickson G. J Neuromuscul Dis. 2021;8(s2):S303-S316. 4. Loboda A, Chamberlain JS, Dulak J. Mol Ther Nucleic Acids. 2025;36(4):102759. 5. Takeda S, Clemens PR, Hoffman EP. J Neuromuscul Dis. 2021;8(s2):S343-S358. 6. Torelli S, Scaglioni D, Sardone V, et al. J Neuropathol Exp Neurol. 2021;80(10):955-965. 7. Brown V, Merikle E, Johnston K, Gooch K, Audhya I, Lowes L. J Patient Rep Outcomes. 2023;7(1):129.