Posts by: Alice Melão

cystic fibrosis, News

Rapid weight gain early in life may have a long-term negative impact on the growth of children with cystic fibrosis (CF), a study has found. This finding suggests that ... Read more

March 27, 2019May 17, 2019

Triple Combo of Proteostasis’ CFTR Modulators May Benefit CF Patients, Phase 1 Trial Shows

cystic fibrosis, News

Treatment with Proteostasis Therapeutics’ triple combo therapy regimen may significantly improve the lung function of patients with cystic fibrosis (CF), results from a Phase 1 clinical trial suggest. The ... Read more

March 25, 2019May 15, 2019

Targeting Bacteria Cell Wall Elements Can Fight Chronic P. aeruginosa Infection, Study Suggests

cystic fibrosis, News

Targeting some elements in the bacterial cell wall can be an effective way to activate the immune system and fight chronic, treatment-resistant Pseudomonas aeruginosa infections, according to a study. ... Read more

March 8, 2019March 26, 2019

Positive Data from VX-445 Triple Combo Regimen Prompt Vertex to Seek Regulatory Approval

News

Vertex Pharmaceuticals is planning to request regulatory approval from the U.S. Food and Drug Administration (FDA) and the European Medicines Agency (EMA) during the third and fourth quarters of 2019, ... Read more

March 6, 2019March 19, 2019

Translate Bio Advancing MRT5005 Through Ongoing Phase 1/2 Trial in CF Patients

cystic fibrosis, News

An ongoing Phase 1/2 clinical trial exploring Translate Bio’s investigational therapy MRT5005 will start testing multiple ascending doses (MAD) in patients with cystic fibrosis (CF). This follows an assessment ... Read more

March 4, 2019March 19, 2019

European HIT-CF Project to Explore ELX-02 as Treatment for Rare CF Mutations

cystic fibrosis, News

Eloxx Pharmaceuticals will explore the potential of its lead investigational drug candidate, ELX-02, as a treatment for patients with cystic fibrosis (CF) caused by rare nonsense genetic mutations, in new ... Read more

February 22, 2019March 12, 2019

New Gene Therapy Strategy May Reverse Effects of Disease-related Mutations, Study Shows

cystic fibrosis

A new strategy to genetically prevent disease-related mutations from occurring shows the potential to treat several genetic diseases, including cystic fibrosis (CF), a study reports. This newly developed approach ... Read more

February 18, 2019February 28, 2019

Vertex Plans to Ask for Extended Approval of Symkevi in Europe for CF Children Ages 6-11

cystic fibrosis, News

Results from a Phase 3 trial demonstrate that Symkevi (tezacaftor and ivacaftor combo), known as Symdeko in the U.S., is safe and can effectively improve lung clearance in children, ... Read more

February 5, 2019February 14, 2019

Group Publishes Guidelines for Home Oxygen Therapy Use in Children

cystic fibrosis

The American Thoracic Society (ATS) recommends the use of home oxygen therapy in children with cystic fibrosis (CF) who have mild or severely reduced oxygen saturation, according to the ... Read more

January 29, 2019February 6, 2019

Toddlers Age 1-2 With CF Can Now Be Treated With Kalydeco in Canada

cystic fibrosis, News

Health Canada has approved the use of Kalydeco (ivacaftor) in toddlers age 1-2 years with certain genetic mutations that prevent the correct functioning of the CFTR protein, the therapy’s developer ... Read more

Posts by: Alice Melão

Alice Melão

Early, Rapid BMI Increase May Affect Overall Growth of CF Children, Study Suggests

Triple Combo of Proteostasis’ CFTR Modulators May Benefit CF Patients, Phase 1 Trial Shows

Targeting Bacteria Cell Wall Elements Can Fight Chronic P. aeruginosa Infection, Study Suggests

Positive Data from VX-445 Triple Combo Regimen Prompt Vertex to Seek Regulatory Approval

Translate Bio Advancing MRT5005 Through Ongoing Phase 1/2 Trial in CF Patients

European HIT-CF Project to Explore ELX-02 as Treatment for Rare CF Mutations

New Gene Therapy Strategy May Reverse Effects of Disease-related Mutations, Study Shows

Vertex Plans to Ask for Extended Approval of Symkevi in Europe for CF Children Ages 6-11

Group Publishes Guidelines for Home Oxygen Therapy Use in Children

Toddlers Age 1-2 With CF Can Now Be Treated With Kalydeco in Canada

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