Duchenne muscular dystrophy newborn screening, a case study for examining ethical and legal issues for pilots for emerging disorders: Considerations and recommendations

Michele A. Lloyd-Puryear, Thomas O. Crawford, Amy Brower, Kristin Stephenson, Tracy Trotter, Edward Goldman, Aaron Goldenberg, R. Rodney Howell, Annie Kennedy, Michael Watson

Research output: Contribution to journalReview article

Abstract

Duchenne muscular dystrophy (DMD/Duchenne) is one of the ten most severe and common pediatric genetic diseases and affects an estimated 1 in every 5000 male births. While Duchenne is a 100% fatal disease, the clinical community has demonstrated that immediate identification and early clinical interventions can add years, even decades to an individual's life span. In anticipation of the changing therapeutic landscape for the Duchenne community, Parent Project Muscular Dystrophy established a newborn screening (NBS) initiative. This initiative included a Bioethics and Legal Issues Workgroup to consider the bioethics and legal issues of NBS for Duchenne. The workgroup's discussion focused only on Duchenne NBS and met through conference calls over a one-year period of time seeking consensus on various identified issues. This article reports on the findings and recommendations from that workgroup.

Original languageEnglish (US)
Article number6
JournalInternational Journal of Neonatal Screening
Volume4
Issue number1
DOIs
StatePublished - Mar 1 2018

Fingerprint

Duchenne Muscular Dystrophy
Ethics
Bioethics
Newborn Infant
Inborn Genetic Diseases
Muscular Dystrophies
Parturition
Pediatrics
Pilots
Therapeutics

Keywords

  • Carrier
  • Duchenne
  • Muscular dystrophy
  • Newborn screening
  • X-linked disease

ASJC Scopus subject areas

  • Pediatrics, Perinatology, and Child Health
  • Obstetrics and Gynecology
  • Immunology and Microbiology (miscellaneous)

Cite this

Duchenne muscular dystrophy newborn screening, a case study for examining ethical and legal issues for pilots for emerging disorders : Considerations and recommendations. / Lloyd-Puryear, Michele A.; Crawford, Thomas O.; Brower, Amy; Stephenson, Kristin; Trotter, Tracy; Goldman, Edward; Goldenberg, Aaron; Rodney Howell, R.; Kennedy, Annie; Watson, Michael.

In: International Journal of Neonatal Screening, Vol. 4, No. 1, 6, 01.03.2018.

Research output: Contribution to journalReview article

Lloyd-Puryear, Michele A. ; Crawford, Thomas O. ; Brower, Amy ; Stephenson, Kristin ; Trotter, Tracy ; Goldman, Edward ; Goldenberg, Aaron ; Rodney Howell, R. ; Kennedy, Annie ; Watson, Michael. / Duchenne muscular dystrophy newborn screening, a case study for examining ethical and legal issues for pilots for emerging disorders : Considerations and recommendations. In: International Journal of Neonatal Screening. 2018 ; Vol. 4, No. 1.
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