bannerHON
img
HONnews
HONnews
img PATIENT / PARTICULIER img PROFESSIONNEL DE SANTE img WEBMESTRE img
img
 
img
HONcode sites
Khresmoi - new !
HONselect
News
Conferences
Images

Themes:
A B C D E F G H I
J K L M N O P Q
R S T U V W X Y Z
Browse archive:
2018: N O S A J J M A M F J
2017: D N

 
  Other news for:
Muscular Diseases
Nervous System Diseases
Spinal Diseases
 Resources from HONselect
Drug for Spinal Muscular Atrophy May Help Older Children: Study

By Robert Preidt

WEDNESDAY, Aug. 29, 2018 (HealthDay News) -- A drug used to treat a rare and deadly muscle-wasting disease in children still improves muscle control even if treatment begins at a later age, a new study found.

Spinal muscular atrophy (SMA) is a leading genetic cause of death in infants worldwide. It attacks nerve cells in the spinal cord, leading to muscle weakness, which can affect breathing, swallowing, walking and head control.

SMA type 1 is the most common and severe form of the disease. It starts before 6 months of age, with infants never gaining the ability to sit up. The median survival rate is eight to 14 months. That means half have shorter lives, half longer.

Previous studies focused on children 7 months old or younger.

This new study included 33 children, ages 8 months to 8 years, with SMA type 1. They were given an injection of the drug nusinersen (Spinraza) into the spinal canal.

The drug increases production of a protein essential for motor neurons in the spinal cord to survive. It was approved by the U.S. Food and Drug Administration in December 2016, and is the first and only approved treatment for all types of SMA.

Six months after the injection, all 33 children had significant muscle control improvements, even the oldest, the researchers reported.

Five patients, ranging from 18 months to 4 years of age when they got their first injection, were able to sit up without support for the first time.

The study was published Aug. 29 in the online issue of the journal Neurology.

"This study is exciting because we found participants had motor function improvement six months after receiving treatment, even an 8-year-old participant," study author Dr. Laurent Servais said in a journal news release.

Servais is a pediatric neurologist at Pitie-Salpetriere Hospital in Paris, France, and Citadelle Hospital in Liege, Belgium.

"The overall response was to the same extent as that in the previously studied younger population," he noted.

"Spinal muscular atrophy type 1 is a devastating disease, and it is encouraging to see that nusinersen may also help people who are at a later stage in the disease process," Servais said.

More research is needed, he said, to find out if genetic factors affect differences in patients' response to treatment.

More information

The Muscular Dystrophy Association has more on spinal muscular atrophy.

SOURCE: Neurology, news release, Aug. 29, 2018

Copyright © 2018 HealthDay. All rights reserved. URL:http://consumer.healthday.com/Article.asp?AID=737152

Resources from HONselect: HONselect is the HON's medical search engine. It retrieves scientific articles, images, conferences and web sites on the selected subject.
Muscular Atrophy
Muscular Atrophy, Spinal
Atrophy
Therapeutics
Muscles
Affect
Neurons
The list of medical terms above are retrieved automatically from the article.

Disclaimer: The text presented on this page is not a substitute for professional medical advice. It is for your information only and may not represent your true individual medical situation. Do not hesitate to consult your healthcare provider if you have any questions or concerns. Do not use this information to diagnose or treat a health problem or disease without consulting a qualified healthcare professional.
Be advised that HealthDay articles are derived from various sources and may not reflect your own country regulations. The Health On the Net Foundation does not endorse opinions, products, or services that may appear in HealthDay articles.


Home img About us img MediaCorner img HON newsletter img Site map img Ethical policies img Contact