Drug Discovery and Development

  • Home Drug Discovery and Development
  • Drug Discovery
  • Women in Pharma and Biotech
  • Oncology
  • Neurological Disease
  • Infectious Disease
  • Resources
    • Video features
    • Podcast
    • Voices
    • Webinars
  • Pharma 50
    • 2025 Pharma 50
    • 2024 Pharma 50
    • 2023 Pharma 50
    • 2022 Pharma 50
    • 2021 Pharma 50
  • Advertise
  • SUBSCRIBE

New Nusinersen Drug Delivery Method Identified for Spinal Muscular Atrophy

By Clinic for Special Chidren | August 27, 2018

A new report has identified an alternative method to deliver nusinersen to patients with spinal muscular atrophy (SMA) using a subcutaneous intrathecal catheter system (SIC) configured by connecting an intrathecal catheter to an implantable infusion port. SMA is a devastating genetic disease that leads to progressive degeneration of motor neurons that control movement, swallowing, and breathing. It is the leading genetic cause of infant death worldwide. Nusinersen is the first FDA approved therapy for SMA but must be administered into the cerebrospinal fluid by repeat lumbar puncture every 4 months for life. Unfortunately, the majority of surviving SMA patients have skeletal deformities or spinal hardware that make it difficult to safely and reliably access the cerebrospinal fluid.

The study, by clinicians and researchers at the Clinic for Special Children in Strasburg, PA and the Nemours/A.I. duPont Hospital for Children in Wilmington DE, appears in the Journal of Pediatric Orthopaedics. Ten SMA patients underwent implantation of the catheter device and received nusinersen dosing through the SIC. The device implantation took less than two hours and was well tolerated in all patients, with an average hospital stay of less than 55 hours. Once the SIC system was implanted, all subsequent nusinersen doses were administered in less than 20 minutes, requiring only topical anesthetic in an outpatient setting. SIC implantation significantly reduced the cost of drug administration.

This SIC dosing method might also benefit SMA patients without advanced neuromuscular disease or spinal pathology; repeated lumbar puncture can prove challenging for any SMA patient, especially newborns and young children, who have the highest rate of traumatic complications (20-50%) when nusinersen is administered by lumbar puncture.

While the study’s observations suggest the SIC system to be relatively safe and without complications, use of the device warrants further multicenter trials. If proven effective in future studies, this method could double the number of patients able to receive nusinersen worldwide and reduce costs five- to ten-fold.

SOURCE: Clinic for Special Children


Filed Under: Neurological Disease

 

Related Articles Read More >

Human study tracks brain’s glymphatic flow in real time, opening Alzheimer’s drug avenues
An 8-year-old patient with gangliosidosis demonstrates improved mobility, walking unassisted on a soccer field after treatment with N-acetyl-L-leucine. (Still from video footage; parental consent obtained for use).
Modified amino acid approved for Niemann–Pick shows promise in early Parkinson’s research
AI-guided hunt points to PHGDH as an upstream drug target in Alzheimer’s disease
Why smaller, simpler molecular glues are gaining attention in drug discovery
“ddd
EXPAND YOUR KNOWLEDGE AND STAY CONNECTED
Get the latest news and trends happening now in the drug discovery and development industry.

MEDTECH 100 INDEX

Medtech 100 logo
Market Summary > Current Price
The MedTech 100 is a financial index calculated using the BIG100 companies covered in Medical Design and Outsourcing.
Drug Discovery and Development
  • MassDevice
  • DeviceTalks
  • Medtech100 Index
  • Medical Design Sourcing
  • Medical Design & Outsourcing
  • Medical Tubing + Extrusion
  • Subscribe to our E-Newsletter
  • Contact Us
  • About Us
  • R&D World
  • Drug Delivery Business News
  • Pharmaceutical Processing World

Copyright © 2025 WTWH Media LLC. All Rights Reserved. The material on this site may not be reproduced, distributed, transmitted, cached or otherwise used, except with the prior written permission of WTWH Media
Privacy Policy | Advertising | About Us

Search Drug Discovery & Development

  • Home Drug Discovery and Development
  • Drug Discovery
  • Women in Pharma and Biotech
  • Oncology
  • Neurological Disease
  • Infectious Disease
  • Resources
    • Video features
    • Podcast
    • Voices
    • Webinars
  • Pharma 50
    • 2025 Pharma 50
    • 2024 Pharma 50
    • 2023 Pharma 50
    • 2022 Pharma 50
    • 2021 Pharma 50
  • Advertise
  • SUBSCRIBE