New ethical challenges in the management of rare pediatric diseases with innovative therapies.

[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.