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August 8, 2019August 8, 2019

CF Foundation Earmarks Up to $15M for Arcturus Therapeutics to Advance Experimental RNA Therapy

cystic fibrosis, News

The Cystic Fibrosis Foundation (CFF) will give up to $15 million to Arcturus Therapeutics to advance the development of an experimental RNA therapy, called LUNAR-CF, to treat CF patients with all ... Read more

August 5, 2019August 5, 2019

Translate Bio’s MRT5005 May Improve Lung Function With a Single Dose, Interim Trial Data Shows

cystic fibrosis, News

A single dose of MRT5005, Translate Bio‘s first-in-class messenger RNA (mRNA) therapy for the treatment of cystic fibrosis (CF), improves patients’ lung function, interim data from a Phase 1/2 ... Read more

August 2, 2019August 2, 2019

Long-term Treatment with Kalydeco Decreases Risk of Lung Infection, Study Reports

cystic fibrosis, News

Treatment for three years with Kalydeco (ivacaftor) reduces the risk of lung infection by serious pathogens — including Pseudomonas aeruginosa and Staphylococcus aureus — in people with cystic fibrosis (CF), a ... Read more

August 1, 2019

Rare Disease Groups Seek Public Support to Renew Newborn Screening Act in Senate

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Screening newborns for genetic diseases with treatments that can prevent crippling or deadly progression, especially for rare disorders, has a ways to go in the United States. No state ... Read more

August 1, 2019August 1, 2019

Using Nebulizer While Lying on Alternating Sides Can Better Deposit Therapy Across Lungs, Trial Finds

cystic fibrosis, News

People using a nebulizer while lying on their side showed no significant loss in a treatment’s overall deposition in the lungs, a study in healthy adults and in cystic ... Read more

July 31, 2019July 31, 2019

Development Bank of Wales Raises Funds for UK’s Cystic Fibrosis Trust

cystic fibrosis, News

Employees at the Development Bank of Wales, in the U.K., have raised £30,400 (around $37,000) to support the Cystic Fibrosis Trust. Every year, bank employees are asked to nominate ... Read more

July 30, 2019July 31, 2019

Proteostasis Begins Dosing in Phase 2 Trial of 3 CF Investigational Therapies

cystic fibrosis, News

The first patient has been dosed in a Phase 2 clinical trial testing Proteostasis Therapeutics‘s combinations of three investigational therapies for cystic fibrosis (CF), the company announced. The three ... Read more

July 29, 2019July 29, 2019

Vertex Names Reshma Kewalramani to Succeed Leiden as CEO, Advance Therapies to 90% of CF Patients

cystic fibrosis, News

Jeffrey Leiden, who in seven years has taken Vertex Pharmaceuticals to the cusp of developing treatments for about 90% of people with cystic fibrosis (CF), is exchanging his day-to-day leadership ... Read more

July 26, 2019July 26, 2019

Arts, Education, Song, and Advocacy Programs Awarded 2019 Impact Grants by CFF

cystic fibrosis, News

Five programs helping to empower the cystic fibrosis (CF) community were selected to receive up to $10,000 each under the fourth annual Impact Grants awarded by the Cystic Fibrosis ... Read more

July 25, 2019July 25, 2019

Mylan Given Rights to Market TOBI Solution and TOBI Podhaler in UK

cystic fibrosis, News

The European Commission (EC) and the U.K.’s Medicines and Healthcare products Regulatory Agency (MHRA) granted Mylan the right to market TOBI Podhaler (tobramycin inhalation powder) and TOBI Solution (tobramycin nebulizer solution) ... Read more

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