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Start Over Descriptor "Cystic fibrosis -- Care and treatment -- Genetic aspects" Topic business, international
10 results on '"Cystic fibrosis -- Care and treatment -- Genetic aspects"'

2. European Commission Approves KAFTRIO[R] (ivacaftor/tezacaftor/elexacaftor) in Combination With Ivacaftor to Treat Children With Cystic Fibrosis Ages 6 to 11 Years

Subjects
Vertex Pharmaceuticals Inc., Cystic fibrosis -- Care and treatment -- Genetic aspects, Drug approval, Ivacaftor, Business, Business, international, European Union. European Commission
Abstract

- More than 1,500 children now eligible to receive a treatment targeting the underlying cause of cystic fibrosis for the first time - LONDON -- Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) [...]

Published
2022

3. Kaftrio Treated Severe CF Without Confirmed Secondary CFTR Mutation

Subjects
Vertex Pharmaceuticals Inc., Genetic research -- Genetic aspects, Cystic fibrosis -- Care and treatment -- Genetic aspects, Pharmaceutical industry, Ivacaftor, Business, international, Law
Abstract

Kaftrio successfully improved lung function and quality of life in three Italian cystic fibrosis (CF) patients who have severe lung disease with an unknown mutation in the CFTR gene in [...]

Published
2021

5. Vertex Announces European Commission Approval for SYMKEVI[R] (tezacaftor/ivacaftor) With KALYDECO[R] (ivacaftor) for Eligible Children With Cystic Fibrosis Ages 6-11 Years

Subjects
Vertex Pharmaceuticals Inc., Cystic fibrosis -- Care and treatment -- Genetic aspects, Ivacaftor, Tezacaftor, Pharmaceutical industry, Drug approval, Business, Business, international, Kalydeco (Medication)
Abstract

- The combination therapy is a new treatment option for CF patients who are homozygous for F508del - - The only medicine to treat the underlying cause of CF in [...]

Published
2020

6. Vertex Announces European Commission Approval for KALYDECO[R] (ivacaftor) as First and Only CFTR Modulator to Treat Eligible Infants With Cystic Fibrosis as Early as Four Months of Age

Subjects
Vertex Pharmaceuticals Inc., Cystic fibrosis -- Care and treatment -- Genetic aspects, Ivacaftor, Pharmaceutical industry, Drug approval, Business, Business, international, Kalydeco (Medication)
Abstract

- Approval provides opportunity to treat the underlying cause of cystic fibrosis earlier than ever before in Europe - LONDON -- Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced that the [...]

Published
2020

7. Vertex Announces European Commission Approval for KALYDECO (ivacaftor) for Children and Adolescents With Cystic Fibrosis Between the Ages of 6 Months and 18 Years With the R117H Mutation in the CFTR Gene

Subjects
Vertex Pharmaceuticals Inc., Cystic fibrosis -- Care and treatment -- Genetic aspects, Ivacaftor, Genes -- Genetic aspects, Pharmaceutical industry, Drug approval, Business, international, Law, European Union. European Commission, Kalydeco (Medication)
Abstract

Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced that the European Commission has granted approval of the label extension for KALYDECO (ivacaftor) to include the treatment of children and adolescents with [...]

Published
2020

8. Vertex Announces European Commission Approval for KALYDECO[R] (ivacaftor) for Children and Adolescents With Cystic Fibrosis Between the Ages of 6 Months and 18 Years With the R117H Mutation in the CFTR Gene

Subjects
Vertex Pharmaceuticals Inc., Cystic fibrosis -- Care and treatment -- Genetic aspects, Ivacaftor, Pharmaceutical industry, Drug approval, Fibrosis, Business, Business, international, Kalydeco (Medication)
Abstract

- KALYDECO([R]) (ivacaftor) is the first and only approved medicine in Europe to treat the underlying cause of cystic fibrosis in patients with the R117H mutation, the most common residual [...]

Published
2020

9. Vertex Receives European CHMP Positive Opinion for KALYDECO[R] (ivacaftor) for Children and Adolescents With Cystic Fibrosis Between the Ages 6 Months and 18 Years With the R117H Mutation in the CFTR Gene

Subjects
Vertex Pharmaceuticals Inc., Cystic fibrosis -- Care and treatment -- Genetic aspects, Ivacaftor, Pharmaceutical industry, Fibrosis, Business, Business, international, Kalydeco (Medication)
Abstract

- If approved, KALYDECO([R]) (ivacaftor) will be the first and only medicine in Europe to treat the underlying cause of cystic fibrosis in patients with the R117H mutation, the most [...]

Published
2020

10. Copernicus to Receive Increased Support from Cystic Fibrosis Foundation Therapeutics to Further Development of its Non-Viral Gene Therapy for Cystic Fibrosis

Subjects
Copernicus Therapeutics Inc., Cystic Fibrosis Foundation Therapeutics Inc., Genetic research -- Genetic aspects, Cystic fibrosis -- Care and treatment -- Genetic aspects, Gene therapy -- Genetic aspects, Biotechnology industry -- Genetic aspects, Business, Business, international
Abstract

CLEVELAND -- Copernicus Therapeutics, Inc., announced today it will receive up to $5.2 million from Cystic Fibrosis Foundation Therapeutics, Inc. (CFFT) to support continued development of a potential gene therapy [...]

Published
2007

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