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Page 247 Angelini. J Transl Genet Genom 2024;8:244-8 https://dx.doi.org/10.20517/jtgg.2024.29

Furthermore, managing effective follow-up and preventing contractures are important components of
optimal care for DMD patients. Some DMD patients develop joint contractures; in such cases, optimal
physiotherapy is beneficial over surgical interventions. Newer treatments, such as deflazacort or
vamorolone, week-end regimens, are also used to accommodate caregivers and families.

Additionally, many clinicians recommend continuing glucocorticoid therapy even after the loss of
ambulation, while others prefer discontinuing it if the patient is wheelchair-bound. Treatment should be
initiated before any physical decline is observed and after thorough family discussions and planning . This
[7-8]
[9]
planning should involve dietary advice, blood tests, and consideration of possible side effects .
The benefits of steroid treatment are more significant with early initiation, which requires an early diagnosis
to prevent cardio-respiratory complications from bracing and overnight ventilators.

The involvement of skeletal and cardiac muscles does not occur simultaneously, but treatment at both levels
is necessary. With the use of assisted ventilation, spinal surgery, and cardiomyopathy management through
drugs, DMD patients can survive up to 40 years.

Regarding Becker muscular dystrophy, which presents with a heterogeneous clinical profile, its treatment is
under investigation . Notably, heart transplants have been performed for both DMD and BMD patients.
[10]
Recent progress in the dystrophinopathy field has been remarkable, and we commend all researchers for
their significant contributions to the field of DMD. I sincerely invite these pioneer scientists to contribute to
a Special Issue of the journal JTGG, which maintains rigorous peer-review standards.

The socioeconomic impact of these disorders is profound, affecting the quality of life and daily activities of
individuals, families and caregivers. Academic achievements in Dystrophinopathy are widely recognized for
their substantial societal and media impact, as well as their engagement with Patient Associations. Once
again, I express my gratitude to researchers for their contributions to Dystrophinopathy research and invite
submissions of case reports, reviews, or exceptional translational research.

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