Pharmacist Digest

4DMT’s CF gene therapy posts early cues for improving patient outcomes

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).

Diabetes

31 Patient and Provider Groups Warn that Mifepristone Ruling Threatens All FDA-approved Drugs

Putting Patients First Blog

APRIL 11, 2023

The Leukemia & Lymphoma Society WomenHeart The post 31 Patient and Provider Groups Warn that Mifepristone Ruling Threatens All FDA-approved Drugs appeared first on National Health Council. We support the FDA’s role in safeguarding patients and urge the Fifth Circuit Court of Appeals to act swiftly to reverse the judge’s decision.

Diabetes

House of Representatives’ Work Requirements Policy Proposal Threatens Healthcare for Millions of Americans

Putting Patients First Blog

APRIL 17, 2023

Komen The AIDS Institute The Leukemia & Lymphoma Society WomenHeart The post House of Representatives’ Work Requirements Policy Proposal Threatens Healthcare for Millions of Americans appeared first on National Health Council.

Hospitals

Insmed trumpets mid-stage results from rare lung disease drug

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).

Immunity

Immunity Immunization

52 Patient Groups Applaud Justice Department’s Stay Request in Braidwood Lawsuit

Putting Patients First Blog

APRIL 13, 2023

Komen The AIDS Institute The Leukemia & Lymphoma Society U=U plus WomenHeart The post 52 Patient Groups Applaud Justice Department’s Stay Request in Braidwood Lawsuit appeared first on National Health Council.

Diabetes

Diabetes Immunity Immunization

Vertex builds in gene editing yet again, with $1.2bn Arbor deal

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.

Diabetes

South Africans take on big pharma for access to ‘miracle’ cystic fibrosis drug

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

Pharmaceutical Companies Pharmaceutical Companies Hospitals

Five Things To Know About Beta-Lactam Therapeutic Drug Monitoring (TDM)

ID Stewardship

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

Nursing Homes Hospitals Dosage

How eligible patient numbers affect price of rare disease drugs in France

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.

Drug Pricing

Drug Pricing Drug Development

Leading Patient Organizations Call for Immediate Action to Protect Patients’ Medicaid Coverage

Putting Patients First Blog

JULY 31, 2023

Komen The AIDS Institute The Leukemia & Lymphoma Society The post Leading Patient Organizations Call for Immediate Action to Protect Patients’ Medicaid Coverage appeared first on National Health Council.

Immunity

Immunity Immunization

Using RWE in rare disease drug development: effective innovations with historical controls

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.

Drug Development

Drug Development Pharmaceutical Companies Pharmaceutical Companies Documentation

A history of the pharmaceutical industry

pharmaphorum

SEPTEMBER 1, 2020

The contraceptive pill, introduced in 1960, had an impact on society almost as massive as that of penicillin, enabling women to effectively control their fertility and enabling sexual equality for the first time. The list of novel drugs from the post-war era speaks for itself.

Diabetes

Diabetes Immunity Immunization Vaccines

Creating an effective patient engagement set of practices that is enduring and future-proof

pharmaphorum

DECEMBER 22, 2021

As an ‘agency’ that generates insights services that bridge the relationships between patients and industry, we are in a unique position to ensure these practices are ethical and productive – ultimately delivering improved outcomes for patients, pharma and society.

Communication

Pharmacist Digest

4DMT’s CF gene therapy posts early cues for improving patient outcomes

31 Patient and Provider Groups Warn that Mifepristone Ruling Threatens All FDA-approved Drugs

Trending Sources

House of Representatives’ Work Requirements Policy Proposal Threatens Healthcare for Millions of Americans

Insmed trumpets mid-stage results from rare lung disease drug

52 Patient Groups Applaud Justice Department’s Stay Request in Braidwood Lawsuit

Vertex builds in gene editing yet again, with $1.2bn Arbor deal

South Africans take on big pharma for access to ‘miracle’ cystic fibrosis drug

Five Things To Know About Beta-Lactam Therapeutic Drug Monitoring (TDM)

How eligible patient numbers affect price of rare disease drugs in France

Leading Patient Organizations Call for Immediate Action to Protect Patients’ Medicaid Coverage

Using RWE in rare disease drug development: effective innovations with historical controls

A history of the pharmaceutical industry

Creating an effective patient engagement set of practices that is enduring and future-proof

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