rare-disease Videos
10 videos found
MPS IVA, also known as Morquio A Syndrome, is a rare disease that affects major organs in the body. Learn more about Morquio A Syndrome from the ...
Mar. 10, 2021
ByBioMarin
One photographer is helping make special memories for parents of children with rare diseases. “I hope to show the world that babies – and people - with special needs are beautiful and ...
Jun. 4, 2022
Together, we aspire to positively impact the lives of those living with rare diseases. ...
Mar. 10, 2021
ByBioMarin
In this video, you will learn more about Philipp’s rare disease and how it has affected his life so far. “Rare", however, is actually not that rare ...
Mar. 10, 2021
Learn how blood-brain barrier modeling is shaping neurological research and how enzyme replacement therapy offers hope for rare diseases. This video also highlights breakthroughs in infectious disease pathogen detection, solutions for Eustachian tube dysfunction, and the latest in commercial orthopedic medical devices. ...
Mar. 12, 2025
You can try Jeeva for 30 days at no-cost cost if: ~You want to recruit more patients faster ~Double documentation is wasting your time ~Your studies require updated features that other tools don't have Promotions: ~Groups that subscribe with Jeeva in the next 2 months receive special pricing on single and institutional licenses ~Rare disease foundations and ...
Apr. 26, 2022
Due to high development costs, diseases with smaller market share, such as rare diseases are only satisfied in 5% of cases leaving nearly 350mln people worldwide without any treatments. ...
Apr. 16, 2021
20 years of rare opportunities. 20 years of personal impact. Despite being faced with potential demise during the company’s infancy, BioMarin persevered to become one of the world leaders in developing therapies for rare genetic diseases. ...
Mar. 10, 2021
ByBioMarin
BioMarin is a global biotechnology company focused on developing first-in-class and best-in-class therapeutics that provide meaningful advances to patients who live with serious and life-threatening rare genetic ...
Mar. 10, 2021
ByBioMarin
Introduction & Industry Update Janet Lambert, CEO, Alliance for Regenerative Medicine Emile Nuwaysir, Ph.D., CEO, Ensoma; Chairman, Alliance for Regenerative Medicine (ARM); Chairman, BlueRock Therapeutics 2022: A Banner Year for Rare Genetic Disease Therapies? Bob Smith, Senior Vice President, Global Gene Therapy Business, Pfizer Inc. ...
May. 30, 2022