Table 10.

Treatment of Manifestations in Individuals with Late-Onset of Sandhoff Disease

Manifestation/ConcernTreatmentConsiderations/Other
Weakness /
Impaired mobility 		PT/OT
  • Adaptive equipment & mobility assists
  • Knee braces may be of particular help for locking knees.
Spasticity/Tremor Symptom-targeted pharmacotherapy by experienced neurologist
Dysarthria / Communication needs By speech-language pathologist
  • Focus on strategies to slow speech rate.
  • Consider eval for alternative means of communication (e.g., AAC) for those w/expressive language difficulties.
Occupational
counseling 		Vocational rehab
Psychiatric issues
  • Antidepressant or antipsychotic medications may be used, but clinical response is variable & can be poor.
  • Cognitive behavioral therapy can ↑ coping skills.
Treatment needs to be individualized.
Family support In-home nursing & respite careCould be indicated for persons w/advanced disease

AAC = augmentative and alternative communication; OT = occupational therapy; PT = physical therapy

From: Sandhoff Disease

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