TRIALS: A Fight for Children with Niemann-Pick Type C, a Fatal Genetic Disease
Descrição
Children with Niemann-Pick Type C (NPC), their parents and scientists seeking a treatment for this fatal genetic disease. WSJ follows this 6-year fight.
What is Niemann-Pick Type C Disease?
Niemann-Pick Disease Type C
The complexity of a monogenic neurodegenerative disease: More than two decades of therapeutic driven research into Niemann-Pick type C disease - ScienceDirect
A Second Chance at Life”: Can Gene Therapies Beat Rare Disease?
Rare Disease to Inform Ebola Susceptibility?
Genes, Free Full-Text
Parents of twins with Niemann-Pick find a treatment
Experimental drug shows promise in cruel childhood disease
mRNA Treatment Rescues Niemann–Pick Disease Type C1 in Patient Fibroblasts
March 2022 – NNPDF
Severely impaired CTL killing is a feature of the neurological disorder Niemann-Pick disease type C1 - ScienceDirect
Parents of twins with 'children's Alzheimer's' find hope in sugar compound therapy
When Drug Development for Rare Disease Hit Setback, Parents Were Stung - WSJ
de
por adulto (o preço varia de acordo com o tamanho do grupo)
