Background: Botulinum toxin is commonly used to treat movement disorders, but is rarely applied
in muscular dystrophies due to concerns about excessive muscle weakening. A clinical
case involving a LAMA2-related muscular dystrophy patient showed promising results
following botulinum toxin injection, prompting further investigation. The aim of this
review was to examine the safety and effectiveness of botulinum toxin injections in
two specific populations: children with torticollis and patients with muscular dystrophies.
The goal was to explore the existing evidence and potential research gaps of botulinum
toxin use as a treatment option in these groups.
Methods: A scoping review according to the PRISMA-ScR guidelines was carried out. Separate
searches for both objectives were performed in the database Ovid MEDLINE. All relevant
studies published up to December 2024 were screened for eligibility by two people.
The data extraction included study characteristics, demographics, disease, and treatment-specific
data.
Results: Thirteen studies (227 paediatric torticollis cases) and ten studies (35 muscular
dystrophy cases) were included. In the majority of children with torticollis, botulinum
toxin improved the range of motion and posture, with few mild side effects. Most cases
of muscular dystrophy showed symptom improvement. Rare cases of excessive muscle weakening
were reported in association with dysphagia.
Conclusion: Botulinum toxin is a promising and mostly safe treatment in children with torticollis
and cases of muscular dystrophy when used cautiously. Further, higher-quality research
is required to establish meaningful results suitable for clinical use.