Emerging Therapeutic Strategies for Fragile X Syndrome: Q&A.

Alusi, Ghassan; Berry-Kravis, Elizabeth; Nelson, David; Orefice, Lauren L; Booker, Sam A

Alusi, Ghassan; Berry-Kravis, Elizabeth; Nelson, David; Orefice, Lauren L; Booker, Sam A.

Afiliação

Alusi G; Barts Health NHS Trust, LondonE1 2ES, U.K.
Berry-Kravis E; Department of Pediatrics, Neurological Sciences, and Anatomy and Cell Biology, Rush University Medical Center, Chicago, Illinois60612, United States.
Nelson D; Molecular and Human Genetics, Baylor College of Medicine, Houston, Texas77030, United States.
Orefice LL; Department of Molecular Biology, Massachusetts General Hospital, Boston, Massachusetts02114, United States.
Booker SA; Department of Genetics, Harvard Medical School, Boston, Massachusetts02115, United States.

ACS Chem Neurosci ; 13(24): 3544-3546, 2022 12 21.

Article em En | MEDLINE | ID: mdl-36475635

ABSTRACT

ABSTRACT

Understanding how best to treat aspects of Fragile X syndrome has the potential to improve the quality of life of affected individuals. Such an effective therapy has, as yet, remained elusive. In this article, we ask those researching or affected by Fragile X syndrome their views on the current state of research and from where they feel the most likely therapy may emerge.

Assuntos

Síndrome do Cromossomo X Frágil; Humanos; Síndrome do Cromossomo X Frágil/tratamento farmacológico; Proteína do X Frágil da Deficiência Intelectual/genética; Qualidade de Vida

Palavras-chave

Fragile X syndrome; arbaclofen; fragile X messenger ribonucleoprotein 1; neurodevelopmental conditions; phosphodiesterase; somatosensation

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Texto completo: 1 Coleções: 01-internacional Base de dados: MEDLINE Assunto principal: Síndrome do Cromossomo X Frágil Limite: Humans Idioma: En Revista: ACS Chem Neurosci Ano de publicação: 2022 Tipo de documento: Article País de afiliação: Reino Unido

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