Skip to content
Pompe Disease News logo
  • About Pompe
    What is Pompe disease?
    Types
    Symptoms
    Causes
    Diagnosis
    Treatment options
    • Lumizyme
    • Nexviazyme
    • Pombiliti + Opfolda
  • News
  • Columns
    Powering Through Pompe — Keara Engle
    Archived columns
    • The Pompe Community Diaries – Anthony Wu
    • On the Road to Pompe — Dwayne Wilson
  • Featured topics
    Living with Pompe
    • Pompe service dog
    • Adaptive clothing
    • Living with Pompe disease
    • ERT and enzyme stabilizers
    • The ERT process
    • Your daily routine with LOPD
    • Adaptive equipment
    • Late-onset Pompe disease
    • Dwayne’s story
    • Expert voice: Safe and effective exercise
    • View all
    Pompe pathways
    • ERT home infusions
    • Therapeutic crossroads
    • Tips for caregivers
    • Maximizing doctor’s appointments
    • Building an LOPD care team
    • Financial planning with Pompe
    • Work and Pompe
    • Occupational therapy
    • Pompe and pain
  • FAQs
  • Advocacy partners
  • What can we help you find today?

August 21, 2019 News by Larry Luxner

Amicus CEO John Crowley Outlines Progress in Pompe, Fabry, Batten Therapies

Pharmaceutical executives rarely make for a sympathetic Hollywood medical drama. But John Crowley did, and in the nearly 10 years since the release of “Extraordinary Measures” — a tearjerker starring Brendan Fraser as Crowley and Harrison Ford as short-tempered scientist Robert Stonehill — biotech has seen a huge transformation, both…

February 26, 2019 News by Larry Luxner

WODC 2019 Organizers Expect 1,200 to Attend Rare Disease Conference in April

The world’s biggest gathering of rare disease researchers, patient groups, pharmaceutical executives, and government officials is planned for April 10–12 in a Washington, D.C., suburb. Some 1,200 people have already registered to attend the World Orphan Drug Congress (WODC) USA 2019, set to take place at the Gaylord National Harbor…

Recent Posts

  • Orthodontic care benefits young girl with infantile Pompe disease
  • Myozyme may help LOPD patients walk, breathe over long term
  • Genetic test flags Pompe disease in expectant father with no symptoms


Envelope icon

Subscribe to our newsletter

Get regular updates to your inbox.

Bionews, Inc.

3 W Garden St
Suite 700
Pensacola, FL 32502
Website: bionews.com
Email: [email protected]
Phone: 1-800-936-1363

  • About Us
  • Leadership
  • Our Culture
  • Editorial Policy
  • Advertising Policy
  • Corrections Policy
  • Terms of Service
  • Privacy Policy
  • Careers
  • Contact Us
Disclaimer

This site is strictly a news and information website about the disease. It does not provide medical advice, diagnosis or treatment. This content is not intended to be a substitute for professional medical advice, diagnosis, or treatment. Always seek the advice of your physician or other qualified health provider with any questions you may have regarding a medical condition. Never disregard professional medical advice or delay in seeking it because of something you have read on this website.

Copyright © 2013-2025 All rights reserved.