Miller TM, Pestronk A, David W, Rothstein J, Simpson E, Appel SH, Andres PL, Mahoney K, Allred P, Alexander K, Ostrow LW, Schoenfeld D, Macklin EA, Norris DA, Manousakis G, Crisp M, Smith R, Bennett CF, Bishop KM, Cudkowicz ME. An antisense oligonucleotide against SOD1 delivered intrathecally for patients with SOD1 familial amyotrophic lateral sclerosis: a phase 1, randomised, first-in-man study. Lancet Neurol. 2013 May;12(5):435-42. Epub 2013 Mar 29 PubMed.

Recommends

Please login to recommend the paper.

Comments

No Available Comments

Make a Comment

To make a comment you must login or register.

This paper appears in the following:

News

  1. Paper Alert: Antisense Oligonucleotide Therapy Safe for ALS?
  2. In Adult Mice, Reduced Tau Quiets Agitated Neurons
  3. Lou Gehrig’s RNA Interference Success in Mice, Monkeys
  4. Second Study Confirms Antisense Oligonucleotides Bust RNA Aggregates
  5. Cloak and Dagger Clusters? How C9ORF72 Repeats Kill Is Still a Mystery
  6. First Round of FTD Therapeutics Fell Short, But Many More Are Up and Running