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Editorial
. 2023 May;67(5):337-338.
doi: 10.1002/mus.27802. Epub 2023 Mar 9.

Keeping expanded access programs in context

James B Caress  1
Affiliations
Editorial

Keeping expanded access programs in context

James B Caress. Muscle Nerve. 2023 May.
No abstract available

Keywords: FDA approval; amyotrophic lateral sclerosis; biomarker; clinical trial; expanded access program.

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Comment on

  • Safety and activity of anti-CD14 antibody IC14 (atibuclimab) in ALS: Experience with expanded access protocol.
    Gelevski D, Addy G, Rohrer M, Cohen C, Roderick A, Winter A, Carey J, Scalia J, Yerton M, Weber H, Doyle M, Parikh N, Kane G, Ellrodt A, Burke K, D'Agostino D, Sinani E, Yu H, Sherman A, Agosti J, Redlich G, Charmley P, Crowe D, Appleby M, Ziegelaar B, Hanus K, Li Z, Babu S, Nicholson K, Luppino S, Berry J, Baecher-Allan C, Paganoni S, Cudkowicz M. Gelevski D, et al. Muscle Nerve. 2023 May;67(5):354-362. doi: 10.1002/mus.27775. Epub 2022 Dec 30. Muscle Nerve. 2023. PMID: 36533976

References

REFERENCES

    1. Jerome RN, Edwards TL, Boswell HC, Bernard GR, Harris PA, Pulley JM. Recommendations to facilitate expanded access to investigational therapies for seriously ill patients. Acad Med. 2016;91:305-309.
    1. Speers MA. Providing patients with critical or life-threatening illnesses access to experimental drug therapy: a guide to clinical trials and the US FDA expanded access program. Pharm Med. 2019;33:89-98.
    1. 1 USC 321, 331, 351, 352, 353,355 360bbb, 371; 42 USC 262. Subpart I-expanded access to investigational drugs for treatment use. Updated November 29, 2022. https://www.accessdata.fda.gov/scripts/cdrh/cfdocs/cfcfr/CFRSearch.cfm?C.... Accessed December 27, 2022.
    1. Abe K, Aoki M, Tsuji S, et al. Safety and efficacy of edaravone in well defined patients with amyotrophic lateral sclerosis: a randomised, double-blind, placebo-controlled trial. Lancet Neurol. 2017;16:505-512.
    1. Paganoni S, Macklin EA, Hendrix S, et al. Trial of sodium phenylbutyrate-taurursodiol for amyotrophic lateral sclerosis. N Engl J Med. 2020;383:919-930.

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