Drug Discovery World

APRIL 22, 2024

The headline news this week all concerns clinical trials for new drugs that could have a significant impact on future patient treatment, including trials testing therapeutics for ALS, cystic fibrosis, gonorrhoea, TB and Parkinson’s.

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Drug Discovery World

NOVEMBER 27, 2023

A label expansion for Kaftrio (ivacaftor/tezacaftor/elexacaftor) in a combination regimen with ivacaftor for the treatment of cystic fibrosis (CF) has been approved in the European Union (EU). The post EU extends paediatric use of combination therapy in cystic fibrosis appeared first on Drug Discovery World (DDW).

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STAT News

FEBRUARY 20, 2023

Dan Brickey was stunned when he learned the news. The cost of the cystic fibrosis medicine that his 2-year-old daughter, Ali, had recently started taking was about to climb from just $180 out of pocket each year to a whopping $43,600 in 2023.

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Drug Discovery World

OCTOBER 14, 2022

News round-up for 10-14 October by DDW Digital Content Editor Diana Spencer. The top news in the world of drug discovery this week reflects the vital importance of collaboration, partnership and support within this industry. . Partnership seeks to transform Cystic Fibrosis research .

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Drug Discovery World

JANUARY 27, 2023

News round-up for 23-27 January by DDW Digital Content Editor Diana Spencer. Cell and gene therapies have been making the news this week, as we launch our very own guide to global innovation in CGT with the Winter issue of DDW, as well as an exciting new virtual event on the same topic.

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The Pharma Data

DECEMBER 4, 2020

Molly Gibson was 6 pounds, 13 ounces, when she was born in October to Tina Gibson and her husband, Ben, T he New York Times reported. The Gibsons decided to use donated embryos because Ben has cystic fibrosis and Tina is a carrier, and they feared passing the disease on to their children, The Times reported. More News Resources.

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XTalks

AUGUST 19, 2020

Social Distancing and Cystic Fibrosis. Cystic fibrosis (CF) patients must keep a six feet distance from others at all times to keep germs away that could exacerbate their disease. Wildlife has been rebounding in nature and appearing all over the news, reminding us about the impact we have on their natural habitats.

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pharmaphorum

NOVEMBER 11, 2021

The five highest health gain categories looked at in the report are: Cystic fibrosis (CFTR modulators, ivacaftor, lumacaftor/ ivacaftor and tezacaftor/ ivacaftor). The good news? Mostly England’s performance improved from 2019 to 2020 and tops the chart for cystic fibrosis medicines. The bad news?

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The Pharma Data

APRIL 14, 2021

Expanding treatment options for personalised care and self-management is always welcome news for the patient community,” said Kenneth Mendez, CEO and President, Asthma and Allergy Foundation of America. alfa) for cystic fibrosis; and Esbriet ® ?(pirfenidone) pirfenidone) for idiopathic pulmonary fibrosis (IPF).

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The Pharma Data

DECEMBER 3, 2020

A member of staff that suffers with Cystic Fibrosis asked the company to make a face mask using our antibacterial fabric. As a result, his CF Consultant requested a supply for his patients, we subsequently donated these to the hospital and also the Cystic Fibrosis Care Charity. The AB Mask was created in July 2019.

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pharmaphorum

OCTOBER 27, 2020

That decision leaves the Cambridge, Massachusetts biotech with a preclinical-stage drug for cystic fibrosis and just over $52 million in cash as it considers its future. Analyst Hartaj Singh at Oppenheimer said there is now “a lack of clarity on the way forward.”.

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pharmaphorum

APRIL 16, 2021

Because we knew that Orphan Medicines, led by the gene therapy Zolgensma and the Cystic Fibrosis treatment Trikafta, had in many cases actually outperformed expectations in 2020, we assessed the first six months of sales for New Active Substance launches with and without Orphan medicines.

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Drug Discovery World

APRIL 17, 2024

A cystic fibrosis drug targeting the basic defect that causes the condition has been shown to be safe and effective in newborns aged four weeks and above, new research has found. The drug, Vertex Pharmaceuticals’ Ivacaftor (Kalydeko), is the first drug designed to treat the basic defect in cystic fibrosis.

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pharmaphorum

FEBRUARY 28, 2022

The positive news is that the pipeline of treatments for rare diseases is healthy, with 40% of new medicines in development being orphan drugs, a spokesperson for the European Federation of Pharmaceutical Industries and Associations (EFPIA) told pharmaphorum.

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pharmaphorum

NOVEMBER 27, 2020

A new EMA approval has expanded the use of Vertex Pharma’s exon-skipping cystic fibrosis therapy Symkevi to children as young as six if they have specific gene mutations. The post Vertex gets EU okay to treat younger cystic fibrosis patients appeared first on.

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Drug Discovery World

JUNE 19, 2023

Anoat Therapeutics’ goal is to bring a transformative and mutation-agnostic treatment option to CF patients for whom existing therapies are not effective – for example, patients not eligible or responsive to CFTR (Cystic Fibrosis Transmembrane Conductance Regulator) modulator therapies.

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Drug Discovery World

MARCH 23, 2023

UK-based Cystic fibrosis (CF) Syndicate in Antimicrobial Resistance (AMR) has announced a £3 million ($3.6 million) Collaborative Discovery Programme for drug discovery innovators to accelerate the development of new treatments for people with CF.

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pharmaphorum

NOVEMBER 24, 2022

Meanwhile, in the UK alone there are over 10,000 people living with cystic fibrosis (CF). She continued: “We’re looking for proposed solutions that target the vicious cycle of infection, inflammation, and lung-damage, which ideally have broad potential benefits for people with bronchiectasis and cystic fibrosis.

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Drug Discovery World

JANUARY 25, 2023

The European Commission has granted orphan designation for Krystal Biotech’s KB407 for the treatment of cystic fibrosis (CF). The post Gene therapy for cystic fibrosis receives EC orphan drug designation appeared first on Drug Discovery World (DDW).

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Delveinsight

NOVEMBER 26, 2020

Million from Cystic Fibrosis Foundation for inhaled antibiotic. million from the Cystic Fibrosis Foundation in order to develop an inhaled antibiotic for lung infections, which are often fatal in people with cystic fibrosis. Polyphor picks up USD 3.3 Polyphor snagged a USD 3.3 Clancy, M.D.,

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Drug Discovery World

OCTOBER 17, 2022

Partnerships have been forged and renewed to shed light on neuropsychiatric disorders, oncology and cystic fibrosis. This round-up was compiled by DDW’s Diana Spencer and reflects the vital importance of collaboration, partnership and support within this industry.

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Drug Discovery World

OCTOBER 13, 2022

LifeArc, a national medical research charity, is joining the Cystic Fibrosis (CF) Antimicrobial Resistance (AMR) Syndicate in a strategic partnership alongside Cystic Fibrosis Trust and Medicines Discovery Catapult (MDC).

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Scienmag

JULY 21, 2022

Cystic fibrosis is well-known for attacking the lungs through thick blockages of mucus, but those with the condition often also develop diabetes because of the way cystic fibrosis can affect the pancreas.

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pharmaphorum

SEPTEMBER 18, 2020

The larger of the two deals is with Vertex and includes $75 million upfront for a project to apply Moderna’s mRNA and lipid nanoparticle (LNP) to the development of drugs to treat cystic fibrosis, with another $380 million in potential milestone payments.

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Drug Discovery World

JULY 12, 2022

Biotechnology company Destiny Pharma has received an award from the Cystic Fibrosis Foundation to help the company advance its lead compound for cystic fibrosis patients who have been infected with methicillin-resistant? Being able to treat MRSA infections effectively could significantly help cystic fibrosis patients.?

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Scienmag

JANUARY 24, 2022

Cystic fibrosis, for instance, is caused by mutations in the CFTR gene that prevent the eponymous protein from assuming its proper configuration. Now, a new study demonstrates that drugs […]. Protein misfolding is a likely culprit in many degenerative disorders.

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Drug Discovery World

OCTOBER 24, 2022

Amid continued concern about antibiotic resistance, the headline news this week was the discovery of a new antibiotic that could be effective against some of the most treatment-resistant pathogens. DDW’s Megan Thomas narrates five key stories of the week to keep DDW subscribers up-to-date on the latest industry updates. .

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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).

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Pfizer

JANUARY 27, 2023

In addition, to learn more, please visit us on www.Pfizer.com and follow us on Twitter at @Pfizer and @Pfizer News , LinkedIn , YouTube and like us on Facebook at Facebook.com/Pfizer. John’s Wort ( hypericum perforatum ) There are limited clinical data available for PAXLOVID.

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Pfizer

DECEMBER 13, 2022

Cystic fibrosis transmembrane conductance regulator potentiators: lumacaftor/ivacaftor. In addition, to learn more, please visit us on www.Pfizer.com and follow us on Twitter at @Pfizer and @Pfizer News , LinkedIn , YouTube and like us on Facebook at Facebook.com/Pfizer. Antimycobacterials: rifampin. Herbal Products: St.

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Pfizer

DECEMBER 20, 2022

Cystic fibrosis transmembrane conductance regulator potentiators: lumacaftor/ivacaftor. In addition, to learn more, please visit us on www.Pfizer.com and follow us on Twitter at @Pfizer and @Pfizer News , LinkedIn , YouTube and like us on Facebook at Facebook.com/Pfizer. Anticancer drugs: apalutamide. Herbal Products: St.

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Pfizer

NOVEMBER 23, 2022

Cystic fibrosis transmembrane conductance regulator potentiators: lumacaftor/ivacaftor. In addition, to learn more, please visit us on www.Pfizer.com and follow us on Twitter at @Pfizer and @Pfizer News , LinkedIn , YouTube and like us on Facebook at Facebook.com/Pfizer. Antimycobacterials: rifampin. Herbal Products: St.

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The Pharma Data

JANUARY 17, 2021

Here’s a look at last week’s news. Johnson & Johnson published interim Phase I/IIa data in the New England Journal of Medicine showing its single-dose COVID-19 vaccine candidate created an immune response that lasted at least 71 days. The year is starting to pick up in terms of clinical trial announcements. COVID-19-Related.

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