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During most eukaryotic translation processes, the small 40S ribosome subunit scans an mRNA from its 5' end until it encounters the first start AUG codon. The large 60S ribosomal subunit then joins the smaller one to initiate protein synthesis. The location of the translation initiation is largely determined by the nucleotides near the start codon as there may be multiple translation initiation sites present on the mRNA.  Marilyn Kozak discovered that the sequence RCCAUGG (where R...
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Development of an Alpha-synuclein Based Rat Model for Parkinson's Disease via Stereotactic Injection of a Recombinant Adeno-associated Viral Vector
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Advancing clinical development for neuronopathic Hunter syndrome through a quantitatively-driven reverse translation

Robert D Latzman1, Olivia Campagne1, Meera E Modi1,2

  • 1Takeda Pharmaceutical Company Limited, Cambridge, Massachusetts, USA.

Clinical and Translational Science
|March 28, 2024
PubMed
Summary
This summary is machine-generated.

Quantitative analysis reveals distinct cognitive trajectories in Hunter syndrome (Mucopolysaccharidosis Type II; MPS-II). Early cognitive scores and biomarkers predict disease progression, aiding rare disease drug development.

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Area of Science:

  • Neuroscience
  • Rare Diseases
  • Drug Discovery

Background:

  • Quantitative evaluation of clinical data accelerates drug discovery but is underutilized, especially for rare diseases.
  • Hunter syndrome (Mucopolysaccharidosis Type II; MPS-II) is a rare, X-linked lysosomal storage disorder with heterogeneous cognitive progression.

Purpose of the Study:

  • To outline a framework for quantitative reverse translation in clinical development.
  • To characterize predictors of cognitive trajectory in MPS-II patients.

Main Methods:

  • Utilized data from 138 MPS-II subjects (age 2.5-10.1 years) from sponsored and natural history studies.
  • Applied mixed-effects models to analyze neurocognitive outcomes and disease progression indicators.
  • Refined target populations based on age, cognitive status, and cerebrospinal fluid glycosaminoglycans (GAG) biomarkers.

Main Results:

  • Identified a specific cognitive development trajectory: initial progression, plateau (4-8 years), then variable decline.
  • Observed faster cognitive decline in subjects with lower baseline cognitive scores.
  • Found accelerated decline associated with higher cerebrospinal fluid GAG levels at enrollment.

Conclusions:

  • Neurocognitive course in MPS-II varies significantly based on age, cognitive function, and biomarker status at enrollment.
  • These findings are crucial for designing more effective clinical trials for MPS-II.
  • The quantitative framework supports improved rare disease drug development.