Posts by: Alice Melão

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

January 28, 2019February 1, 2019

Personalized Nutrition Programs May Help Improve Outcomes for CF Patients, Survey Suggests

cystic fibrosis, News

New personalized strategies that can address the nutritional needs of cystic fibrosis patients may provide the best care and improve patient outcomes, a study suggests. The study, “A survey ... Read more

January 24, 2019February 5, 2019

European Research Team Granted €6.8M to Develop Non-viral Gene Therapy System

cystic fibrosis, News

The Horizon 2020 research and innovation program of the European Union granted €6.8 million (approximately $7.7 million) to an international research team for the development of non-viral gene delivery ... Read more

January 23, 2019January 31, 2019

Orkambi Approved in Europe for Younger CF Patients with F508del Mutations

cystic fibrosis, News

The European Commission has approved the label extension of Orkambi (lumacaftor/ivacaftor) for the treatment of children ages 2-5 with cystic fibrosis (CF) due to F508del mutations, Vertex Pharmaceuticals, the ... Read more

Posts by: Alice Melão

Alice Melão

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

Personalized Nutrition Programs May Help Improve Outcomes for CF Patients, Survey Suggests

European Research Team Granted €6.8M to Develop Non-viral Gene Therapy System

Orkambi Approved in Europe for Younger CF Patients with F508del Mutations

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