Results 91 to 100 of about 5,931 (183)
IBMTR/ABMTR Mortimer M. Bortin Awards for Best Clinical Research Abstracts [PDF]
, 2002 Mortimer M. Bortin, M.D., was one of the founding members of the International Bone Marrow Transplant Registry and served as its Scientific Director for more than 30 years. The Mortimer M. Bortin Award, established to commemorate Dr.
Gri Espinagosa, Josep +1 more
core +1 more source
Atidarsagene autotemcel for metachromatic leukodystrophy [PDF]
, 2023 Metachromatic leukodystrophy (MLD) is a rare autosomal recessive disorder of sphingolipid metabolism, due to a deficiency of the enzyme arylsulfatase A (ARSA).
Gissen, P, Messina, M
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White Matter Disease in a Toddler With New‐Onset Seizures
Annals of the Child Neurology Society, Volume 3, Issue 3, Page 245-246, September 2025.
Olivia Viscuso +3 more
wiley +1 more source
Development of the Impact of Juvenile Metachromatic Leukodystrophy on Physical Activities scale
Journal of Patient-Reported Outcomes, 2018 Background Metachromatic leukodystrophy (MLD) is a rare disease with three forms based on the age at onset of signs and symptoms. The objective of this study was to develop a caregiver-reported clinical outcome assessment that measures impairments in ...
T. Michelle Brown +3 more
doaj +1 more source
We describe a strain of Lassa virus representing a putative new lineage that was isolated from a cluster of human infections with an epidemiologic link to Togo. This finding extends the known range of Lassa virus to Togo.2018UL1 TR000454/TR/NCATS NIH HHS/
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Developing therapeutic approaches for metachromatic leukodystrophy
Drug Design, Development and Therapy, 2013 Shilpa A Patil,1 Gustavo HB Maegawa1,2 1McKusick-Nathans Institute of Genetic Medicine, 2Department of Pediatrics, The Johns Hopkins School of Medicine, Baltimore, MD, USA Abstract: Metachromatic leukodystrophy (MLD) is an autosomal recessive lysosomal ...
Patil SA, Maegawa GHB
doaj
Clinical and Radiological Evaluation of Pediatric Leukodystrophies: A Prospective Study from a Tertiary Care Center in Pakistan [PDF]
Objective: To evaluate the clinical and magnetic resonance imaging (MRI) characteristics of pediatric patients with leukodystrophies and assess the diagnostic value of MRI in a low-resource setting. Materials & Methods: This prospective observational
Inthikhab Khalil +5 more
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Magnetic resonance imaging (MRI) findings in white matter disease of brain [PDF]
, 2008 Demyelinating and dysmyelinating white matter diseases are important components of neurological problems. Recently, Magnetic Resonance Imaging (MRI) has played a key role in diagnoses of white matter diseases.
Ahsan, Humera +5 more
core +1 more source