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:
2017: D N O S A J J M A M F J
2016: D

  Mepsevii Approved for Rare Enzyme Disorder

By Scott Roberts

WEDNESDAY, Nov. 15, 2017 (HealthDay News) -- Mepsevii (vestronidase alfa-vjbk) has been approved by the U.S. Food and Drug Administration to treat a rare, genetic disorder called mucopolysaccharidosis type VII, sometimes called Sly syndrome.

Affecting fewer than 150 people worldwide, the extremely rare disorder typically causes various skeletal abnormalities that worsen with age, including short stature. Life expectancy and symptoms and vary widely, but also may include heart valve problems, enlarged liver and narrowed respiratory airways, the agency said Wednesday in a news release.

Mepsevii is designed to reverse an enzyme deficiency that can lead to the buildup of toxic substances in the body's cells.

"Prior to today's approval, patients with this rare, inherited condition had no approved treatment options," said Dr. Julian Beitz, a director from one of the agency's drug evaluation departments.

Clinical studies of the treatment involved 23 people ranging from 5 months to 25 years old. The most common side effects included allergic-like reactions at the infusion site, diarrhea, rash and severe anaphylaxis reaction.

The treatment is produced by Novato, Calif.-based Ultragenyx Pharmaceutical, which as a condition of approval will conduct additional research to evaluate its long-term safety.

More information

The FDA has more about this approval.

SOURCE: Nov. 15, 2017 press release, U.S. Food and Drug Administration

Copyright © 2017 HealthDay. All rights reserved. URL:http://consumer.healthday.com/Article.asp?AID=728563

Resources from HONselect: HONselect is the HON's medical search engine. It retrieves scientific articles, images, conferences and web sites on the selected subject.
Therapeutics
Heart Valves
Lead
Heart
Drug Evaluation
Evaluation Studies
Liver
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.


Inicio img Sobre nosotros img Rincón de la prensa img Boletín HON img Mapa del sitio img Política ética img Contactos