New ethical challenges in the management of rare pediatric diseases with innovative therapies.
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[1] A. Olry,et al. Estimating cumulative point prevalence of rare diseases: analysis of the Orphanet database , 2019, European Journal of Human Genetics.
[2] The Lancet Diabetes & Endocrinology. Spotlight on rare diseases. , 2019, The lancet. Diabetes & endocrinology.
[3] H. Tabor,et al. Ethical Challenges Confronted When Providing Nusinersen Treatment for Spinal Muscular Atrophy , 2017, JAMA pediatrics.
[4] N. King,et al. New treatments for serious conditions: ethical implications , 2017, Gene Therapy.
[5] The Lancet Neurology. Rare advances for rare diseases , 2017, The Lancet Neurology.
[6] K. Borski. Ethical issues in rare diseases. , 2015, Developmental period medicine.
[7] C. Rodwell,et al. Rare disease policies to improve care for patients in Europe. , 2015, Biochimica et biophysica acta.
[8] Aris Angelis,et al. Socio-economic burden of rare diseases: A systematic review of cost of illness evidence. , 2015, Health policy.
[9] L. Curnow,et al. Receiving Enzyme Replacement Therapy for a Lysosomal Storage Disorder: A Preliminary Exploration of the Experiences of Young Patients and Their Families , 2013, Journal of Genetic Counseling.
[10] J. Dhondt. Expanded newborn screening: social and ethical issues , 2010, Journal of Inherited Metabolic Disease.
[11] H. Tabor,et al. Perspectives on Spinraza (Nusinersen) Treatment Study: Views of Individuals and Parents of Children Diagnosed with Spinal Muscular Atrophy. , 2019, Journal of neuromuscular diseases.
[12] Luis A Barrera,et al. Ethical aspects on rare diseases. , 2010, Advances in experimental medicine and biology.