From Failure to Meet the Clinical Endpoint to U.S. Food and Drug Administration Approval: 15th Antisense Oligonucleotide Therapy Approved Qalsody (Tofersen) for Treatment of <i>SOD1</i> Mutated Amyotrophic Lateral Sclerosis.

van Roon-Mom, Willeke; Ferguson, Chantal; Aartsma-Rus, Annemieke

From Failure to Meet the Clinical Endpoint to U.S. Food and Drug Administration Approval: 15th Antisense Oligonucleotide Therapy Approved Qalsody (Tofersen) for Treatment of SOD1 Mutated Amyotrophic Lateral Sclerosis.

van Roon-Mom, Willeke; Ferguson, Chantal; Aartsma-Rus, Annemieke.

Afiliação

van Roon-Mom W; Department of Human Genetics, Dutch Center for RNA Therapeutics, Leiden University Medical Center, Leiden, The Netherlands.
Ferguson C; Department of Human Genetics, Leiden University Medical Center, Leiden, The Netherlands.
Aartsma-Rus A; RNA Therapeutics Institute, UMass Chan Medical School, Worcester, Massachusetts, USA.

Nucleic Acid Ther ; 33(4): 234-237, 2023 08.

Article em En | MEDLINE | ID: mdl-37581487

Assuntos

Esclerose Lateral Amiotrófica; Oligonucleotídeos Antissenso; Humanos; Esclerose Lateral Amiotrófica/tratamento farmacológico; Esclerose Lateral Amiotrófica/genética; Mutação; Oligonucleotídeos Antissenso/uso terapêutico; Superóxido Dismutase-1/genética; Estados Unidos; United States Food and Drug Administration; Aprovação de Drogas; Determinação de Ponto Final

Texto completo

Imprimir

XML

PubMed Links

Buscar no Google

Texto completo: 1 Base de dados: MEDLINE Assunto principal: Oligonucleotídeos Antissenso / Esclerose Lateral Amiotrófica Idioma: En Ano de publicação: 2023 Tipo de documento: Article

Texto completo

Imprimir

XML

PubMed Links

Buscar no Google