Design of a Randomized, Placebo-Controlled, Phase 3 Trial of Tofersen Initiated in Clinically Presymptomatic SOD1 Variant Carriers: the ATLAS Study

Preventing amyotrophic lateral sclerosis: insights from pre-symptomatic neurodegenerative diseases

(2021) Michael Benatar et al.   BRAIN

Genetic testing for neurodegenerative diseases: Ethical and health communication challenges

(2020) J. Scott Roberts et al.   NEUROBIOLOGY OF DISEASE

Phase 1–2 Trial of Antisense Oligonucleotide Tofersen for SOD1 ALS

(2020) Timothy Miller et al.   NEW ENGLAND JOURNAL OF MEDICINE

Defining pre-symptomatic amyotrophic lateral sclerosis

(2019) Michael Benatar et al.   Amyotrophic Lateral Sclerosis and Frontotemporal Degeneration

Antisense Oligonucleotide Therapies for Neurodegenerative Diseases

(2019) C. Frank Bennett et al.   Annual Review of Neuroscience

Nusinersen initiated in infants during the presymptomatic stage of spinal muscular atrophy: Interim efficacy and safety results from the Phase 2 NURTURE study

(2019) Darryl C. De Vivo et al.   NEUROMUSCULAR DISORDERS

Neurofilaments in pre-symptomatic ALS and the impact of genotype

(2019) Michael Benatar et al.   Amyotrophic Lateral Sclerosis and Frontotemporal Degeneration

Neurofilament light: A candidate biomarker of pre-symptomatic ALS and phenoconversion

(2018) Michael Benatar et al.   ANNALS OF NEUROLOGY

Antisense oligonucleotides extend survival and reverse decrement in muscle response in ALS models

(2018) Alex McCampbell et al.   JOURNAL OF CLINICAL INVESTIGATION

Mutant superoxide dismutase aggregates from human spinal cord transmit amyotrophic lateral sclerosis

(2018) Elaheh Ekhtiari Bidhendi et al.   ACTA NEUROPATHOLOGICA

The Global State of the Genetic Counseling Profession

(2018) MaryAnn Abacan et al.   EUROPEAN JOURNAL OF HUMAN GENETICS

Chemistry, mechanism and clinical status of antisense oligonucleotides and duplex RNAs

(2017) Xiulong Shen et al.   NUCLEIC ACIDS RESEARCH

The path to specialist multidisciplinary care in amyotrophic lateral sclerosis: A population- based study of consultations, interventions and costs

(2017) Miriam Galvin et al.   PLoS One

Defining SOD1 ALS natural history to guide therapeutic clinical trial design

(2016) Taha Bali et al.   JOURNAL OF NEUROLOGY NEUROSURGERY AND PSYCHIATRY

Presymptomatic ALS genetic counseling and testing

(2016) Michael Benatar et al.   NEUROLOGY

Genotype-phenotype correlations of amyotrophic lateral sclerosis

(2016) Hong-Fu Li et al.   Translational Neurodegeneration

Genotype-phenotype relationship in hereditary amyotrophic lateral sclerosis

(2015) Satoshi Yamashita et al.   Translational Neurodegeneration

Delayed diagnosis in ALS: The problem continues

(2014) Hipolito Nzwalo et al.   JOURNAL OF THE NEUROLOGICAL SCIENCES

SOD1 Function and Its Implications for Amyotrophic Lateral Sclerosis Pathology

(2014) Rosie K. A. Bunton-Stasyshyn et al.   NEUROSCIENTIST

Diagnosis pathway for patients with amyotrophic lateral sclerosis: retrospective analysis of the US Medicare longitudinal claims database

(2013) James R Williams et al.   BMC Neurology

Is SOD1 loss of function involved in amyotrophic lateral sclerosis?

(2013) Rachele A. Saccon et al.   BRAIN

Genetic counselling in ALS: facts, uncertainties and clinical suggestions

(2013) A. Chio et al.   JOURNAL OF NEUROLOGY NEUROSURGERY AND PSYCHIATRY

Presymptomatic studies in ALS: Rationale, challenges, and approach

(2012) M. Benatar et al.   NEUROLOGY

Impact of presymptomatic genetic testing for familial amyotrophic lateral sclerosis

(2011) Joanna H Fanos et al.   GENETICS IN MEDICINE

Amyotrophic lateral sclerosis

(2011) Matthew C Kiernan et al.   LANCET

Variation in aggregation propensities among ALS-associated variants of SOD1: Correlation to human disease

(2009) Mercedes Prudencio et al.   HUMAN MOLECULAR GENETICS

Non–cell autonomous toxicity in neurodegenerative disorders: ALS and beyond

(2009) Hristelina Ilieva et al.   JOURNAL OF CELL BIOLOGY

Incidence of amyotrophic lateral sclerosis in Europe

(2009) G. Logroscino et al.   JOURNAL OF NEUROLOGY NEUROSURGERY AND PSYCHIATRY