The Success of the New Drug on Cystic Fibrosis Brings New Hope for Newborns

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A groundbreaking study has revealed the safety and effectiveness of a drug targeting the fundamental defect underlying cystic fibrosis in newborns aged four weeks and older, with a two-year-old boy from Co Meath and his sister playing pivotal roles.

Described as a “significant moment” by one of the lead researchers from RCSI University of Medicine and Health Sciences and Children’s Health Ireland, sister and brother Kara (5) and Isaac Moss (2) both contributed to the study conducted through Children’s Health Ireland.

Kara participated in an earlier phase of the study, which contributed to the approval of the drug for older infants and paved the way for the latest trial involving Isaac. Isaac made history as the first newborn diagnosed with cystic fibrosis globally and was directly enrolled in a trial for these groundbreaking treatments from birth.

Debbie Moss, mother to Kara and Isaac said: ”Both Kara and Isaac are doing really well and remarkably are not experiencing any of the typical symptoms of cystic fibrosis at the moment.”

“Research studies like this one are so important to ensuring that children get access to the right treatments as early as possible. With the right medications, they can enjoy a healthy childhood and look forward to a brighter future.”

Ivacaftor (Kalydeco) represents the first drug specifically designed to target the underlying defect in cystic fibrosis. Initially approved for adults, its authorization extended gradually to encompass older and younger children over several years. Presently, the drug is sanctioned for infants aged four months and above. However, recent research suggests its safety and efficacy in infants as young as four weeks old.

Cystic fibrosis experts emphasize the importance of initiating treatment at an early stage, as it is believed to potentially slow down or halt the progression of the condition in children. This focus on early intervention forms the basis of numerous international research endeavors spearheaded by RCSI and Children’s Health Ireland.

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