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PediNOVA Collaborative

an adult woman holds her baby in a sitting position as it reaches for a toy a woman is holding on a laptop screen

Pediatric Neurodevelopmental Outcomes and Virtual Assessment

The PediNOVA Collab applies a multidisciplinary, measurement-based approach to selection, development, adaptation, validation, and interpretation of neurodevelopmental clinical assessment measures for infants and young children with rare, progressive pediatric neurogenetic conditions. To address the needs of global therapeutic clinical trials, we study the utility of virtual, videoconference-based tools and traditional in person clinical outcome measures. 

 

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Participate in Our Study

We are seeking participation from families of children with infantile-onset neurogenetic conditions who are interested participating in our virtual videoconference study validating a new clinical rating scale.

Participate

Current Research

Our current work centers on refining and evaluating the psychometric properties of our novel infant neurodevelopmental rating scale (the iNDRS) to establish reliability, validity, and responsiveness in infantile-onset rare neurogenetic progressive disease populations including:

  • GM-1 Gangliosidosis
  • GM-2 Gangliosidoses (Tay-Sachs/Sandhoff Diseases)
  • Canavan Disease
  • Adrenoleukodystrophy (ALD)

We are actively engaged in several pediatric rare disease natural history studies and clinical trials evaluating safety and efficacy of therapeutic agents, including:

 to Evaluate Safety, Tolerability, Pharmacokinetics, and Exploratory Efficacy Following ABBV-CLS-7262 Administration in Adult and Pediatric Subjects with vanishing White Matter Disease. NCT05757141. 

MGH PI: Florian Eichler, MD (MGH Neurology/Neurogenetics). ABBVie Pharmaceuticals.

to Evaluate the Efficacy, Safety, Pharmacokinetics and Pharmacodynamics of Intrathecally Administered ION373 in Patients with Alexander Disease (AxD). NCT04849741. 

MGH PI: Florian Eichler, MD (MGH Neurology). Ionis Pharmaceuticals. 

PI: Patricia Musolino, MD, PhD (MGH Neurology). 

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