Significant Progress Made in Fighting Cystic Fibrosis-But More Work Needs to Be Done – Interview

Significant Progress Made in Fighting Cystic Fibrosis-But More Work Needs to Be Done – Interview

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May is Cystic Fibrosis Awareness Month



May is Cystic Fibrosis (CF) Awareness Month and it is a time to celebratethe progress made to advance the care for people with CF but also to acknowledge the work that lies ahead.

Cystic fibrosis (CF) is a life-shortening, progressive, rare genetic disease that leads to multi-organ damage. Given CF is a genetic disease, people are born with it, making it critically important to seek treatment early to potentially slow the progression of this devastating disease.

There are about 88,000 people living with CF worldwide, with about 32,000 in the US.

CF presents a variety of symptoms including frequent coughing, coughing up mucus, shortness of breath, recurring infections, inflammation, and digestion issues.

The FDA recently approved expanded use for a CF treatment to include children ages 2 through 5 years old. This marks an important milestone — more than 900 additional children with CF can now benefit from the medication. The FDA also approved an expansion of another CF medication to include babies 1 to 4 months old.

While significant advances have been made,

there still is more work to ensure all people living with CF have treatments.

Join me in a recent interview with Fred Van Goor, Vice President of CF Research at Vertex as he discussed cystic fibrosis, the need for continued research, and the latest treatments.

See the entire interview here:  Cystic Fibrosis – MichiganMamaNews – YouTube

For more information, go to


Fredrick Van Goor, Ph.D.
Vice President, Cystic Fibrosis Research and Head of Cystic Fibrosis Research Program
Vertex Pharmaceuticals

Fredrick Van Goor, Ph.D., is the Vice President of Cystic Fibrosis (CF) Research for Vertex Pharmaceuticals.  In this role, he works with the CF Disease Area Team to drive the company’s research, clinical, regulatory and commercial strategy and execution.

Dr. Van Goor led the biology effort for the team that discovered and developed Kalydeco, Orkambi, Symdeko, and Trikafta – Vertex’s four approved small molecule medicines that treat the underlying cause of disease in up to 90% of people with CF.  Currently, he is leading Vertex’s collaboration with Moderna to discover novel CFTR mRNA therapies for the treatment of CF.

To date, the collaboration has led to the discovery of VX-522, which is in early-stage clinical studies in people with CF.  For his contributions to CF drug discovery, Dr. Van Goor has been awarded the Wiley Prize (2023), Boomer Esiason Foundation Jerry Cahill Legacy Award (2019), Respiratory Innovation Award (2018), and Doris F. Tulcin Research Award for outstanding accomplishments in CF research (2013).

Dr. Van Goor has over 20 US patents in the areas of CF and technology development and has published six book chapters in the areas of CF, ion channel drug discovery and endocrinology and authored more than 50 research articles in peer-reviewed journals including the New England Journal of Medicine, Nature Medicine, Proceedings of the National Academy of Science, Journal of Neuroscience, Journal of Biological Chemistry and Molecular Pharmacology. Prior to joining Vertex in 2001, Dr. Van Goor was a Postdoctoral Fellow at the National Institutes of Health. Dr. Van Goor received his Ph.D. in Biological Sciences from the University of Alberta, Canada.

Interview courtesy: Vertex Pharmaceuticals

Cynthia Tait

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