source: shutterstock.com

ICYMI: Kanuma for Lysosomal Acid Lipase Deficiency Now Approved in England

Post author:Jessica Lynn
Post published:January 15, 2024
Post category:Lysosomal Acid Lipase Deficiency Wolman disease

In 2015, the United States approved Kanuma (sebelipase alfa) for the treatment of infants living with rare lysosomal acid lipase deficiency. However, the therapy was not approved in other countries;…

Share Your Story

February is RARE DISEASE MONTH! Patient Worthy believes that every rare story deserves to be told. Click below to tell us yours.

We believe rare disease patients are people, not a diagnosis. Through education, awareness and some humor, we help patients, caregivers and support persons by providing relevant and often inspirational news and stories.

Our goals are to share stories, cultivate strong community, provide the latest medical findings, connect people and pioneer production of patient worthy information. Help us attain these goals by telling us a little bit about yourself!

Share Your Story

ICYMI: Kanuma for Lysosomal Acid Lipase Deficiency Now Approved in England

Metastatic Breast Cancer: Navigating Grief

Rethinking What It Means to Live With Acromegaly

The Let’s Chat CAR T One-on-One Mentor Program: Speaking with Someone Who Understands What You Are Going Through

Share Your Story

Let’s Work Together!

Keep Up to Date

Learn More