Article Text
Statistics from Altmetric.com
In his 1973 book ‘Small Is Beautiful’, the economist EF Schumacher argued that technological development and economic growth should be centred on the needs of individuals and communities, and that bigger (unregulated expansion) was not necessarily better.1 We can make a similar case regarding highly technical interventions to manage people with chronic and progressive neurological disorders, including degenerative ataxia. Spectacular advances in genomics appear to offer incredible opportunities to influence the natural histories of ‘untreatable’ conditions, using technologies such as gene silencing/editing and the therapeutic use of agents such as antisense oligonucleotides. However, these treatments, even if proven effective, will be affordable for only a small proportion of patients worldwide. By focusing on these potentially dramatic treatments, patients (and their families) might be left feeling that only technically advanced and expensive interventions will be beneficial, with consequent therapeutic nihilism. Can we do more for our patients with what we already have?
For patients with ataxia (and probably for all neurodegenerative conditions), there are …
Footnotes
Contributors I am the sole author. I was invited to write this piece by the editors of Practical Neurology, following the peer review of a paper submitted to the journal. The concepts explored here have been developed and moulded by conversations with other neurologists, physicians and occupational therapists.
Funding The author has not declared a specific grant for this research from any funding agency in the public, commercial or not-for-profit sectors.
Competing interests None declared.
Provenance and peer review Commissioned. Internally peer reviewed.
Linked Articles
- Editors’ commentary
- Image of the moment
Read the full text or download the PDF:
Other content recommended for you
- Cerebellar degeneration in primary Sjӧgren syndrome
- Integrative network and brain expression analysis reveals mechanistic modules in ataxia
- A kindred with cerebellar ataxia and thermoanalgesia
- Republished: A practical approach to late-onset cerebellar ataxia: putting the disorder with lack of order into order
- A practical approach to late-onset cerebellar ataxia: putting the disorder with lack of order into order
- Clinical assessment of the sensory ataxias; diagnostic algorithm with illustrative cases
- Molecular pathology of ataxia telangiectasia
- The autosomal dominant spinocerebellar ataxias: emerging mechanistic themes suggest pervasive Purkinje cell vulnerability
- Causes of progressive cerebellar ataxia: prospective evaluation of 1500 patients
- A homozygous mutation of VWA3B causes cerebellar ataxia with intellectual disability