Enrollment Stopped in 2 Trials of Treatments for CF Lung Infections
Savara Pharmaceuticals has stopped patient enrollment into two of its ongoing clinical trials in cystic fibrosis (CF) patients — AVAIL and ENCORE — for ... Read more
Updates
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ReCode Therapeutics Raises $80M to Boost Development of CF Therapies
ReCode Therapeutics has raised $80 million in Series A financing to support the preclinical development of its lead targeted RNA therapy for cystic fibrosis (CF). ... Read more -
MRT5005 Gets FDA Rare Pediatric Disease Status as Adult Trial Faces Delay Due to COVID-19
Translate Bio‘s therapy MRT5005 has been given a rare pediatric disease designation by the U.S. Food and Drug Administration (FDA) for the treatment ... Read more -
I’m Holding a Funeral for My Former Self
I am at a loss with loss. Yesterday, I lost my last remaining telecommuting job, my dance company (in the sense that we are ... Read more -
Vertex Assures Supply of CF Therapies, But COVID-19 Likely to Affect Trials
Vertex Pharmaceuticals is “highly confident” that it can continue to supply its approved cystic fibrosis (CF) therapies to patients worldwide, but expects at least some of its ongoing ... Read more
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Trikafta Therapy Prompted the Most Unexpected Emotions
The excitement and flurry around the new combination therapy Trikafta (elexacaftor/tezacaftor/ivacaftor) brought a lot ... -
Pulmonary Fibrosis Patient Spearheads Lung Transplant Foundation to Help Others
At 3 a.m. on July 6, 2003, Miami resident Jeffrey Goldstein got a call ... -
Eloxx Pauses Phase 2 Trials of ELX-02 in Cystic Fibrosis Due to COVID-19
Eloxx Pharmaceuticals has temporarily paused enrollment in Phase 2 clinical trials of ELX-02 in ... -
I’m Worried About Coughing in Public
Lately, I’ve had a lot of fears. Like others in the global cystic fibrosis ... -
NORD Partners with ‘Sing Me a Story’ for July 18–20 Living Rare Forum
The Living Rare, Living Stronger Patient and Family Forum, originally set for May 14–16 ... -
Problems with CFTR Protein in Platelets Linked to Lung Inflammation, Injury
Problems with the CFTR protein in platelets, blood cells essential to clotting, activated these cells, ...
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