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UnknownInborn errors of energy metabolism associated with myopathies.
Not available
S-EPMC2877206 | biostudies-literature
UnknownDietary habits and metabolic control in adolescents and young adults with phenylketonuria: self-imposed protein restriction may be harmful.
Not available
S-EPMC4110337 | biostudies-literature
UnknownHepatic sinusoidal obstruction syndrome and short-term application of 6-thioguanine in pediatric acute lymphoblastic leukemia.
Not available
S-EPMC8410596 | biostudies-literature
UnknownNewbornscreening SMA - From Pilot Project to Nationwide Screening in Germany.
Not available
S-EPMC9881029 | biostudies-literature
UnknownImprovements in Walking Distance during Nusinersen Treatment - A Prospective 3-year SMArtCARE Registry Study.
Not available
S-EPMC9881023 | biostudies-literature
UnknownClinical Effectiveness of Newborn Screening for Spinal Muscular Atrophy: A Nonrandomized Controlled Trial.
Not available
S-EPMC11002769 | biostudies-literature
UnknownEfficacy and safety of gene therapy with onasemnogene abeparvovec in children with spinal muscular atrophy in the D-A-CH-region: a population-based observational study.
Not available
S-EPMC11492610 | biostudies-literature
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