Pharmaceutical Technology

JUNE 8, 2023

The use of 4D Molecular Therapeutics’ (4DMT) aerosolised gene therapy 4D-710 has improved the quality-of-life and spirometry-measured outcomes in three cystic fibrosis patients , based on early results from a Phase I/II study presented at this year’s annual meeting of the European Cystic Fibrosis Society (ECFS).

FDA 98

FDA 98

pharmaphorum

SEPTEMBER 9, 2020

US biotech Insmed has trumpeted updated phase 2 data from its brensocatib, at this week’s virtual European Respiratory Society conference, showing the drug prolonged time to exacerbations caused by the rare lung disease known as non-cystic fibrosis bronchiectasis (NCFBE).

Immunization 59

Immunization FDA 59

pharmaphorum

AUGUST 24, 2021

It’s not the first time Vertex and Arbor have worked together – in 2018, they collaborated on a project focused on genetic diseases, including cystic fibrosis, Vertex’ core therapeutic focus with four approved therapies that treat around 90% of CF patients.

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52

IDStewardship

NOVEMBER 26, 2023

Patient characteristics that may be associated with alterations in beta-lactam PK/PD include critical illness, extremes of age or weight, organ dysfunction ( e.g. , renal impairment or augmented renal clearance), and hypermetabolic disease states such as cystic fibrosis (see Figure 1). Guilhaumou R, Benaboud S, Bennis Y et al.

Nursing Homes 237

Nursing Homes Hospitals Dosage 237

pharmaphorum

DECEMBER 7, 2022

However, these prices bear little risk to the Society Security ( Securité Sociale ) in France as lower CEPS-negotiated prices are subject to payback of all or part of the difference. This is because ATUc prices are not negotiated and are determined by drug manufacturers for the duration of both ATUc and post-ATUc periods.

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105

The Guardian - Pharmaceutical Industry

MARCH 17, 2024

Vertex makes a “miracle drug” called Trikafta that can transform the lives of people with cystic fibrosis. The medication gives them a normal life expectancy, rather than facing the likelihood of dying as young adults, and lives that are no longer blighted by frequent lung infections and hospital admissions.

Pharmaceutical Companies 77

Pharmaceutical Companies Pharmaceutical Companies Hospitals 77

European Pharmaceutical Review

SEPTEMBER 22, 2022

4 For example, the Early Pseudomonal Infection Control randomised trial described the efficacy of different antibiotic regimens for eradication of newly identified Pseudomonas (Pa) in children with cystic fibrosis (CF). Standard Care versus Protocol Based Therapy for New Onset Pseudomonas aeruginosa in Cystic Fibrosis.

FDA 109

FDA Pharmaceutical Companies Pharmaceutical Companies Documentation 109