Defining SOD1 ALS natural history to guide therapeutic clinical trial design

In vivo kinetic approach reveals slow SOD1 turnover in the CNS

(2015) Matthew J. Crisp et al.   JOURNAL OF CLINICAL INVESTIGATION

Antisense Oligonucleotide Therapy for the Treatment of C9ORF72 ALS/FTD Diseases

(2014) Giulietta Riboldi et al.   MOLECULAR NEUROBIOLOGY

Adeno-associated Virus–mediated Delivery of a Recombinant Single-chain Antibody Against Misfolded Superoxide Dismutase for Treatment of Amyotrophic Lateral Sclerosis

(2014) Priyanka Patel et al.   MOLECULAR THERAPY

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

(2013) Timothy M Miller et al.   LANCET NEUROLOGY

Pyrimethamine decreases levels of SOD1 in leukocytes and cerebrospinal fluid of ALS patients: A phase I pilot study

(2012) Dale J. Lange et al.   Amyotrophic Lateral Sclerosis and Frontotemporal Degeneration

SOD1 in Cerebral Spinal Fluid as a Pharmacodynamic Marker for Antisense Oligonucleotide Therapy

(2012) Leah Winer et al.   JAMA Neurology

Effects of gender in amyotrophic lateral sclerosis

(2010) Pamela A. McCombe et al.   Gender Medicine

Rate of familial amyotrophic lateral sclerosis: a systematic review and meta-analysis

(2010) S. Byrne et al.   JOURNAL OF NEUROLOGY NEUROSURGERY AND PSYCHIATRY

The natural history of ALS is changing: Improved survival

(2009) Muddasir Qureshi et al.   Amyotrophic Lateral Sclerosis

Practice Parameter update: The care of the patient with amyotrophic lateral sclerosis: Drug, nutritional, and respiratory therapies (an evidence-based review): Report of the Quality Standards Subcommittee of the American Academy of Neurology

(2009) R. G. Miller et al.   NEUROLOGY