Lucy Nizinski

Maya is one of the lucky ones. She was born in the right place, at the right time, and with two copies of the common mutation of the cystic fibrosis (CF) gene, called DeltaF508. When Maya was born, the average life expectancy of a person with CF was 27 years. That was devastating news for new parents to hear. Maya suffered for twenty years through numerous surgeries, hospitalizations and complications as cystic fibrosis slowly wreaked havoc in her lungs, sinuses, pancreas and kidneys. Through it all, we remained positive, and made life as “normal” as possible.

The Cystic Fibrosis Foundation (CFF) has funded most research related to CF treatments and the quest for a cure primarily because there was just not enough upside for big pharma. In 2012, CFF entered into a venture-philanthropy agreement with Vertex Pharmaceuticals which led to a drug called Kalydeco. This was a breakthrough drug that worked stunningly well, but only for about 4% of the CF population who had a specific CF mutation. In February 2018, Symdeko, a double combination drug by Vertex was approved by the FDA for use by patients with one copy of the mutation Maya has. It was somewhat effective but had significant negative side effects. Finally, in the Fall of 2019, Trikafta became available. This was the miracle drug we had waited for so long!

Trikafta is a triple-drug combination drug by Vertex that Maya started on November 23, 2019. To say it was a miracle is an understatement! Maya’s lung function jumped over 10%, she was coughing less, she gained weight, she was less stuffy, she could actually smell, and she could laugh without coughing! This drug was made possible by contributions to the CFF by people like you! Every single dollar ever given to the CF Foundation made this possible. Every single hour/minute of time volunteered, every Great Strides walked, or any form of support made this difference!

Since starting Trikafta, Maya finished her bachelor’s in social work at UNC-Charlotte and completed her Master’s degree in Child Life at the College of Charleston. She is now a Certified Child Life Therapist at the Children’s Hospital of the King’s Daughters in Norfolk, Virginia. She works in Surgery with children and absolutely loves her job!

Trikafta is a life-long medication, it is not a cure. It does have side-effects, and no one knows the effects of long-term use of the drug since it is such a new medication. Further, there is a small portion of the CF population who were left out, who continue to suffer from the devastating effects of this disease. We cannot abandon them! So we continue our efforts to raise money for the CF Foundation and the life-saving research it funds. We are participating in the Great Strides Walk for a cure for CF on September 28, 2024 at The Station at Plaza West, Charlotte NC. If you are able to help by making a donation, we will be forever grateful. Your contributions have made a difference in the past and will continue to in the future. We can’t wait for the day when CF is actually cured or every single person with CF has a miracle drug like Maya!