Results 151 to 160 of about 5,931 (183)

Coverage with evidence development for medicines with insufficient evidence of clinical benefit: experience from the Netherlands. [PDF]

Int J Technol Assess Health Care
Versteeg JW, Stam N, Mantel-Teeuwisse AK, Timmers L, Goettsch W, Leopold C.   +5 more
europepmc   +1 more source

Metachromatic leukodystrophy: A story of hope woven from sorrow. [PDF]

Mol Ther
Adang L.
europepmc   +1 more source

Newborn Screening for Metachromatic Leukodystrophy in Tuscany: The Paradigm of a Successful Preventive Medicine Program. [PDF]

Int J Neonatal Screen
Malvagia S, Bettiol A, Porcaro M, Mura M, Funghini S, Ombrone D, Forni G, Scolamiero E, Coppi F, Damiano R, Cereda C, Simonetti S, Lonetti A, Daniotti M, Caciotti A, Morrone A, Calbi V, Fumagalli F, Aiuti A, Procopio E, Guerrini R, la Marca G.   +21 more
europepmc   +1 more source

Monogenic Mimics of Neuroinflammatory Phenotypes in Children and Young Adults: An Evolving Landscape. [PDF]

Neurol Genet
Gupta A, Sahjwani D, Kahn I, Gombolay GY, Sen K.   +4 more
europepmc   +1 more source

FPIES after umbilical cord blood transplantation: A case series. [PDF]

Pediatr Allergy Immunol
Cuppen L, Schuurhof A, Versluijs B, Bresters D, Bierings M, Stadermann M, Lindemans CA.   +6 more
europepmc   +1 more source

[Metachromatic leukodystrophy].

Revista chilena de pediatria, 1985
M, Aracena, E, Valenzuela, E, Selman, C, Milos   +3 more
openaire   +1 more source

A cohort of allogeneic hematopoietic stem cell transplantation for pediatric rare diseases (allo-HSCT-PRD): design and preliminary results. [PDF]

Ann Hematol
Hu S, Wang P, Li Z, Jiang W, Liu L, Ma W, Huang Y, Wang X, Du X, Qian X, Wang H, Wang Y, Zhang Y, Zhai X.   +13 more
europepmc   +1 more source

Metachromatic Leukodystrophy in Morocco: Identification of Causative Variants by Next-Generation Sequencing (NGS). [PDF]

Genes (Basel)
Hammoud M, Domínguez-Ruiz M, Assiri I, Rodrigues D, Aboussair N, Lanza VF, Villarrubia J, Colón C, Fdil N, Del Castillo FJ.   +9 more
europepmc   +1 more source

Typical and atypical MRI patterns of rare brain disorders: The challenge of low numbers. [PDF]

Dev Med Child Neurol
Wolf NI, Engelen M, van der Knaap MS.
europepmc   +1 more source