Genetic counseling for the dystrophinopathies—Practice resource of the National Society of Genetic Counselors.

The dystrophinopathies encompass the phenotypically variable forms of muscular dystrophy caused by pathogenic variants in the DMD gene. The dystrophinopathies include the most common inherited muscular dystrophy among 46,XY individuals, Duchenne muscular dystrophy, as well as Becker muscular dystrop...

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Publicado en:Journal of Genetic Counseling Vol. 34; no. 1; pp. 1 - 23
Autores principales: Pickart, Angela M., Martin, Ann S., Gross, Brianna N., Dellefave‐Castillo, Lisa M., McCallen, Leslie M., Nagaraj, Chinmayee B., Rippert, Alyssa L., Schultz, Catherine P., Ulm, Elizabeth A., Armstrong, Niki
Formato: review tables/charts Journal Article
Publicado: Wiley-Blackwell Feb2025
Acceso en línea:Ver este registro en EBSCOhost
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      dt: Feb2025
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      pub: Wiley-Blackwell
      place: Malden, Massachusetts
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        atl: Genetic counseling for the dystrophinopathies—Practice resource of the National Society of Genetic Counselors.
      aug:
        au:
          Pickart, Angela M.
          Martin, Ann S.
          Gross, Brianna N.
          Dellefave‐Castillo, Lisa M.
          McCallen, Leslie M.
          Nagaraj, Chinmayee B.
          Rippert, Alyssa L.
          Schultz, Catherine P.
          Ulm, Elizabeth A.
          Armstrong, Niki
        affil: Department of Laboratory Medicine and Pathology, Mayo Clinic, Rochester Minnesota,, USA
      sug:
        subj:
          Muscular Dystrophy, Duchenne
          Genetic Counseling
          Genetic Screening
          Genetic Counselors
          Professional Practice
          Information Resources
          Muscular Dystrophy, Duchenne Diagnosis
          Muscular Dystrophy, Duchenne Therapy
          Clinical Trials
          Muscular Dystrophy, Duchenne Risk Factors
          Muscular Dystrophy, Duchenne Complications
          Muscular Dystrophy, Duchenne Epidemiology
          Diagnosis, Laboratory
          Adrenal Cortex Hormones Therapeutic Use
          Gene Therapy
      ab: The dystrophinopathies encompass the phenotypically variable forms of muscular dystrophy caused by pathogenic variants in the DMD gene. The dystrophinopathies include the most common inherited muscular dystrophy among 46,XY individuals, Duchenne muscular dystrophy, as well as Becker muscular dystrophy and other less common phenotypic variants. With increased access to and utilization of genetic testing in the diagnostic and carrier setting, genetic counselors and clinicians in diverse specialty areas may care for individuals with and carriers of dystrophinopathy. This practice resource was developed as a tool for genetic counselors and other health care professionals to support counseling regarding dystrophinopathies, including diagnosis, health risks and management, psychosocial needs, reproductive options, clinical trials, and treatment. Genetic testing efforts have enabled genotype/phenotype correlation in the dystrophinopathies, but have also revealed unexpected findings, further complicating genetic counseling for this group of conditions. Additionally, the therapeutic landscape for dystrophinopathies has dramatically changed with several FDA‐approved therapeutics, an expansive research pathway, and numerous clinical trials. Genotype–phenotype correlations are especially complex and genetic counselors' unique skill sets are useful in exploring and explaining this to families. Given the recent advances in diagnostic testing and therapeutics related to dystrophinopathies, this practice resource is a timely update for genetic counselors and other healthcare professionals involved in the diagnosis and care of individuals with dystrophinopathies.
      pubtype: Academic Journal
      doctype:
        review
        tables/charts
        Journal Article
      ougenre: Article
    language: English
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