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Parent Experiences of Sanfilippo Syndrome Impact and Unmet Treatment Needs: A Qualitative Assessment.
Porter, Katherine Ackerman; O'Neill, Cara; Drake, Elise; Parker, Samantha; Escolar, Maria L; Montgomery, Stacey; Moon, William; Worrall, Carolyn; Peay, Holly L.
Affiliation
  • Porter KA; Center for Genomics, Bioinformatics, and Translational Research, RTI International, Research Triangle Park, NC, USA. kmporter@rti.org.
  • O'Neill C; Cure Sanfilippo Foundation, Columbia, SC, USA.
  • Drake E; Cure Sanfilippo Foundation, Columbia, SC, USA.
  • Parker S; Patient and Policy Affairs, Lysogene, Neuilly sur Seine, France.
  • Escolar ML; University of Pittsburgh Medical Center, Pittsburgh, PA, USA.
  • Montgomery S; Children's Hospital of Pittsburgh of UPMC, Pittsburgh, PA, USA.
  • Moon W; Cure Sanfilippo Foundation Parent Advocates, Columbia, SC, USA.
  • Worrall C; Cure Sanfilippo Foundation Parent Advocates, Columbia, SC, USA.
  • Peay HL; Cure Sanfilippo Foundation Parent Advocates, Columbia, SC, USA.
Neurol Ther ; 10(1): 197-212, 2021 Jun.
Article in En | MEDLINE | ID: mdl-33263924

Full text: 1 Collection: 01-internacional Database: MEDLINE Type of study: Guideline / Qualitative_research Aspects: Patient_preference Language: En Journal: Neurol Ther Year: 2021 Document type: Article Affiliation country: Country of publication:

Full text: 1 Collection: 01-internacional Database: MEDLINE Type of study: Guideline / Qualitative_research Aspects: Patient_preference Language: En Journal: Neurol Ther Year: 2021 Document type: Article Affiliation country: Country of publication: