Timothy M. Miller

Has gene therapy for ALS arrived?

Proposed modification to data analysis for statistical motor unit number estimate.

Unraveling the mechanisms involved in motor neuron degeneration in ALS.

Toxicity of familial ALS-linked SOD1 mutants from selective recruitment to spinal mitochondria.

Medicine. Treating neurodegenerative diseases with antibiotics.

Virus-delivered small RNA silencing sustains strength in amyotrophic lateral sclerosis.

Muscle cramps.

Should the Babinski sign be part of the routine neurologic examination?

Progressive spinal axonal degeneration and slowness in ALS2-deficient mice.

Antisense oligonucleotide therapy for neurodegenerative disease.

Amyotrophic lateral sclerosis and gene therapy.

The muscular Dystrophy Surveillance Tracking and Research Network (MD STARnet): surveillance methodology.

Gene transfer demonstrates that muscle is not a primary target for non-cell-autonomous toxicity in familial amyotrophic lateral sclerosis.

RCADiA: simple automation platform for comparative multidimensional protein identification technology.

Differential diagnosis of myotonic disorders.

Gene-targeted therapies for the central nervous system.

Selective association of misfolded ALS-linked mutant SOD1 with the cytoplasmic face of mitochondria.

Mutation analysis in a population-based cohort of boys with Duchenne or Becker muscular dystrophy.

Delayed diagnosis in duchenne muscular dystrophy: data from the Muscular Dystrophy Surveillance, Tracking, and Research Network (MD STARnet).

TDP-43 mutant transgenic mice develop features of ALS and frontotemporal lobar degeneration.

Familial ALS with extreme phenotypic variability due to the I113T SOD1 mutation.

Use of corticosteroids in a population-based cohort of boys with duchenne and becker muscular dystrophy.

ALS-linked mutant superoxide dismutase 1 (SOD1) alters mitochondrial protein composition and decreases protein import.

SOD1 in cerebral spinal fluid as a pharmacodynamic marker for antisense oligonucleotide therapy.

An antisense oligonucleotide against SOD1 delivered intrathecally for patients with SOD1 familial amyotrophic lateral sclerosis: a phase 1, randomised, first-in-man study.