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101. Fast Facts: Long-Chain Fatty Acid Oxidation Disorders : Understand, Identify and Support

102. Fast Facts: Les troubles d'oxydation des acides gras à chaîne longue : Comprendre, identifier et aider

103. Fast Facts para pacientes: Distúrbios da oxidação dos ácidos graxos de cadeia longa

104. Fast Facts pour les patients: Les troubles d'oxydation des acides gras à chaîne longue

105. Fast Facts for Patients: Long-Chain Fatty Acid Oxidation Disorders

108. Molecular and Clinical Genetics of Mitochondrial Diseases Due to POLG Mutations

114. 63 - Other Genodermatoses

115. Spectrum of limb disruption defects associated with chorionic villus sampling

116. Efficacy of Sapropterin Dihydrochloride in Increasing Phenylalanine Tolerance in Children with Phenylketonuria: A Phase III, Randomized, Double-Blind, Placebo-Controlled Study

119. Cardiopulmonary Exercise Testing Reflects Improved Exercise Capacity in Response to Treatment in Morquio A Patients: Results of a 52-Week Pilot Study of Two Different Doses of Elosulfase Alfa

127. Consensus guidelines for newborn screening, diagnosis and treatment of infantile Krabbe disease.

129. Impact of Elosulfase Alfa on Pain in Patients with Morquio A Syndrome over 52 Weeks: MOR-008: A Randomized, Double-Blind, Pilot Study

133. Efficacy and safety of intrathecal idursulfase in pediatric patients with mucopolysaccharidosis type II and early cognitive impairment: Design and methods of a controlled, randomized, phase II/III multicenter study

137. Impact of long-term elosulfase alfa on activities of daily living in patients with Morquio A syndrome in an open-label, multi-center, phase 3 extension study

144. Elosulfase alfa treatment and changes in physical functioning and disability in Morquio syndrome type A

145. Impact of Elosulfase Alfa on Pain in Patients with Morquio A Syndrome over 52 Weeks

149. Impact of long-term elosulfase alfa treatment on respiratory function in patients with Morquio A syndrome.

150. Pregnancy in patients with mucopolysaccharidosis: a case series.

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