Treatment for speech disorder in Friedreich ataxia and other hereditary ataxia syndromes

Vogel, Adam P.; Folker, Joanne E; Poole, Matthew L.

doi:10.1002/14651858.cd008953.pub2

Cited by 29 publications

(16 citation statements)

References 60 publications

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“…glycopyrrhonium, trihexyphenidyl) and fluoxetine 7,12 may ameliorate drooling, although they were not included in a recent Cochrane Review on treatment options in hereditary ataxias. 151 Injections of botulinum neurotoxin may be considered if drooling persists after conservative treatment. 152 Videofluoroscopic swallowing studies may be useful to detect silent aspiration; 98 however, one should try to avoid this diagnostic tool to minimize exposure to ionizing radiation.…”

Section: Evidence-and Expert-based Recommendationsmentioning

confidence: 99%

Ataxia‐telangiectasia: recommendations for multidisciplinary treatment

Haaxma

Flier

et al. 2017

Develop Med Child Neuro

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Ataxia-telangiectasia is a rare, neurodegenerative, and multisystem disease, characterized by cerebellar ataxia, oculocutaneous telangiectasia, immunodeficiency, progressive respiratory failure, and an increased risk of malignancies. It demands specialized care tailored to the individual patient's needs. Besides the classic ataxia-telangiectasia phenotype, a variant phenotype exists with partly overlapping but some distinctive disease characteristics. This guideline summarizes frequently encountered medical problems in the disease course of patients with classic and variant ataxia-telangiectasia, in the domains of neurology, immunology and infectious diseases, pulmonology, anaesthetic and perioperative risk, oncology, endocrinology, and nutrition. Furthermore, it provides a practical guide with evidence- and expert-based recommendations for the follow-up and treatment of all these different clinical topics.

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Section: Evidence-and Expert-based Recommendationsmentioning

confidence: 99%

Ataxia‐telangiectasia: recommendations for multidisciplinary treatment

Haaxma

Flier

et al. 2017

Develop Med Child Neuro

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“…In a recent survey by Ataxia UK [2], people with progressive ataxia identified speech and communication problems as one of the top three most troublesome symptoms of their disease with significant negative impact on their lives. Whilst our understanding of the nature of the communication problems experienced by these patients has improved significantly over time [3][4][5][6][7][8][9][10][11][12][13], a Cochrane Review on treatment efficacy for progressive ataxia syndromes concluded that "there is insufficient and low or very low quality evidence from either RCTs or observational studies to determine the effectiveness of any treatment for speech disorder" (Vogel et al, p. 1 [14]). Clinicians are therefore currently unsure of how to deal with ataxic dysarthria.…”

Section: Introductionmentioning

confidence: 99%

Feasibility and Acceptability of Lee Silverman Voice Treatment in Progressive Ataxias

2020

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Communication difficulties have considerable impact on people with progressive ataxia, yet there are currently no evidencebased treatments. LSVT LOUD® focuses on the production of healthy vocal loudness whilst also improving breath support, vocal quality, loudness and articulation in participating patients. This study aimed to investigate whether Lee Silverman Voice Treatment (LSVT LOUD®) can improve communication effectiveness in these patients. We performed a rater-blinded, singlearm study investigating LSVT LOUD® treatment in a population of patients with progressive ataxia including Friedreich's ataxia (n = 18), spinocerebellar ataxia type 6 (n = 1), idiopathic cerebellar ataxia (n = 1), and spastic paraplegia 7 (n = 1). Twenty-one patients were recruited to the study, with 19 completing treatment. Sessions were administered via Skype in the LSVT-X format, meaning two sessions per week over a period of 8 weeks. Assessments included two baseline and two post-treatment measures and focused on outcome measures covering aspects ranging from physiological function to impact and participation. Results indicate improvements in patient-perceived outcomes for 14 of the 19 participants, in both speech and psychosocial domains. Speech data furthermore demonstrate significant improvements in prolonged vowel duration, and voice quality measures. Intelligibility and naturalness evaluations showed no change post-treatment. Patients reported high acceptability of the treatment itself, as well as administration by Skype. This is the largest treatment study for people with progressive ataxia published to date. It provides an indication that LSVT LOUD® can have a positive impact on communication in this patient group and could form the basis for larger-scale trials.

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“…In IDE-treated FA patients, generally, a reduction in oxidative stress markers was reported and many patients showed an improvement of non-neurological symptoms, as well. However, in a subsequent randomized, double-blind, placebo-controlled trial, no statistically significant difference between the placebo and IDE was revealed on the International Cooperative Ataxia Rating Scale [ 9 , 10 ].…”

Section: Introductionmentioning

confidence: 99%

Idebenone: Novel Strategies to Improve Its Systemic and Local Efficacy

et al. 2018

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The key role of antioxidants in treating and preventing many systemic and topical diseases is well recognized. One of the most potent antioxidants available for pharmaceutical and cosmetic use is Idebenone (IDE), a synthetic analogue of Coenzyme Q10. Unfortunately, IDE’s unfavorable physicochemical properties such as poor water solubility and high lipophilicity impair its bioavailability after oral and topical administration and prevent its parenteral use. In recent decades, many strategies have been proposed to improve IDE effectiveness in the treatment of neurodegenerative diseases and skin disorders. After a brief description of IDE potential therapeutic applications and its pharmacokinetic and pharmacodynamic profile, this review will focus on the different approaches investigated to overcome IDE drawbacks, such as IDE incorporation into different types of delivery systems (liposomes, cyclodextrins, microemulsions, self-micro-emulsifying drug delivery systems, lipid-based nanoparticles, polymeric nanoparticles) and IDE chemical modification. The results of these studies will be illustrated with emphasis on the most innovative strategies and their future perspectives.

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Treatment for speech disorder in Friedreich ataxia and other hereditary ataxia syndromes

Abstract: Treatment for speech disorder in Friedreich ataxia and other hereditary ataxia syndromes.

Cited by 29 publications

References 60 publications

Ataxia‐telangiectasia: recommendations for multidisciplinary treatment

Ataxia‐telangiectasia: recommendations for multidisciplinary treatment

Feasibility and Acceptability of Lee Silverman Voice Treatment in Progressive Ataxias

Idebenone: Novel Strategies to Improve Its Systemic and Local Efficacy

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