Preliminary Safety and Tolerability of a Novel Subcutaneous Intrathecal Catheter System for Repeated Outpatient Dosing of Nusinersen to Children and Adults With Spinal Muscular Atrophy.

08:00 EDT 21st August 2018 | BioPortfolio

Summary of "Preliminary Safety and Tolerability of a Novel Subcutaneous Intrathecal Catheter System for Repeated Outpatient Dosing of Nusinersen to Children and Adults With Spinal Muscular Atrophy."

Many patients with spinal muscular atrophy (SMA) who might benefit from intrathecal antisense oligonucleotide (nusinersen) therapy have scoliosis or spinal fusion that precludes safe drug delivery. To circumvent spinal pathology, we designed a novel subcutaneous intrathecal catheter (SIC) system by connecting an intrathecal catheter to an implantable infusion port.


Journal Details

This article was published in the following journal.

Name: Journal of pediatric orthopedics
ISSN: 1539-2570


DeepDyve research library

PubMed Articles [15191 Associated PubMed Articles listed on BioPortfolio]

Safety and tolerability of subcutaneous sarilumab and intravenous tocilizumab in patients with rheumatoid arthritis.

Safety and efficacy of mAbs blocking the IL-6 receptor have been established in RA. This is the first analysis examining safety and tolerability of sarilumab and tocilizumab administered as single or ...

Safety and tolerability of mice to repeated subcutaneous injections of a peptide mix as a potential vaccine against HCV infection.

In this study the safety and tolerability of new candidate HCV vaccine named Cenv6 were screened in mice. Cenv6 peptide is composed of 6 synthetic HCV peptides (3 structural and 3 nonstructural peptid...

Removal of Intrathecal Catheters Used in Drug Delivery Systems.

Implanted intrathecal drug delivery systems (IDDS) are increasingly used in the treatment of spasticity and in patients with refractory pain. Literature discussing complications associated with intrat...

Safety profile of subcutaneous trastuzumab for the treatment of patients with HER2-positive early or locally advanced breast cancer: primary analysis of the SCHEARLY study.

Subcutaneous trastuzumab (H SC) is a valuable alternative to the intravenous formulation. This study assessed H SC safety and tolerability in human epidermal growth factor receptor 2 (HER2)+ early/loc...

Sodium bicarbonate catheter lock solution reduces hemodialysis catheter loss due to catheter-related thrombosis and blood stream infection: an open-label clinical trial.

There is no ideal lock solution that prevents hemodialysis (HD) catheter loss due to catheter-related thrombosis (CRT) and catheter-related bloodstream infection (CRBSI). Catheter loss is associated w...

Clinical Trials [15995 Associated Clinical Trials listed on BioPortfolio]

Prospective SecurAcath Subcutaneous Securement Trial

To prospectively monitor the safety and performance of the catheter securement device in subjects whose catheter is secured with the Interrad Medical SecureAcath device.

Trial on the Impact of Technical Anesthetic in Pregnancies With Intrauterine Growth Restriction

The combined spinal-epidural analgesia during the labor has been associated with changes in fetal heart rate, which the opinions in literature are not fully consolidated and some studies h...

Concurrent Intrathecal-pemetrexed and Involved-field Radiotherapy for Leptomeningeal Metastasis From Solid Tumors

Intrathecal chemotherapy is one of the most important treatment modalities for leptomeningeal metastasis of solid tumors. In the previous retrospective study, it has been proved that concu...

Labour Analgesia; Comparing a Combinations of Either Fentanyl or Bupivacaine With Intrathecal Morphine

This study is aimed at evaluating single short spinal technique as a form of labour analgesia in a setting where more expensive and labor intensive techniques are not feasible.The study wi...

Subcutaneous Bupivacaine Decrease Post-op Pain in Patients Undergoing C-Section

Camparing post-op pain scores and oxycodone usage in patients who receive jsut intrathecal morphine to patients who receive intrathecal morphine plus subcutaneous bupivacaine .25%, to pati...

Medical and Biotech [MESH] Definitions

Freedom from exposure to danger and protection from the occurrence or risk of injury or loss. It suggests optimal precautions in the workplace, on the street, in the home, etc., and includes personal safety as well as the safety of property.

A hindrance to the passage of fluids through a CATHETER.

Infections resulting from the use of catheters. Proper aseptic technique, site of catheter placement, material composition, and virulence of the organism are all factors that can influence possible infection.

Regional infusion of drugs via an arterial catheter. Often a pump is used to impel the drug through the catheter. Used in therapy of cancer, upper gastrointestinal hemorrhage, infection, and peripheral vascular disease.

Placement of a balloon-tipped catheter into the pulmonary artery through the antecubital, subclavian, and sometimes the femoral vein. It is used to measure pulmonary artery pressure and pulmonary artery wedge pressure which reflects left atrial pressure and left ventricular end-diastolic pressure. The catheter is threaded into the right atrium, the balloon is inflated and the catheter follows the blood flow through the tricuspid valve into the right ventricle and out into the pulmonary artery.

Quick Search


DeepDyve research library

Relevant Topics

Pediatrics is the general medicine of childhood. Because of the developmental processes (psychological and physical) of childhood, the involvement of parents, and the social management of conditions at home and at school, pediatrics is a specialty. With ...

Antisense therapy
Most human diseases are caused by production of abnormal proteins or malfunctioning proteins.   Antisense therapy involves inhibiting production of these proteins.  When a gene is known to cause a specific disease and the genetic sequence ...

Arthritis Fibromyalgia Gout Lupus Rheumatic Rheumatology is the medical specialty concerned with the diagnosis and management of disease involving joints, tendons, muscles, ligaments and associated structures (Oxford Medical Diction...

Searches Linking to this Article