Too Old to Operate15d
Effect of elexacaftor-tezacaftor-ivacaftor on liver transient elastography, fibrosis indices and blood tests in children with cystic fibrosis.
Elexacaftor-tezacaftor-ivacaftor (ETI) has significantly improved the clinical course of people with cystic fibrosis ... liver fibrosis indices and liver tests in children with CF aged 6-12 years ...
River Journal17d
New York Medical College Clinical Trial Becomes First to Administer Novel Inhaled mRNA Therapy to a Patient with Cystic Fibrosis in the United States
My body took time to adjust over the holidays, but I am hopeful that 2025 will bring success in advancements in gene therapy for cystic fibrosis. I can’t wait ... inhaled therapy for CF based on mRNA.
The Bakersfield Californian18d
4DMT Focuses Pipeline to Prioritize 4D-150 in Wet AMD & DME and 4D-710 in CF and Extends ...
Strategically focused pipeline optimizes resource allocation to progress two product candidates with strongest clinical proof of concept: 4D-150 for wet AMD and DME and 4D-710 for cystic fibrosis ...
emjreviews.com18d
mRNA-Based T-Cell Therapy Offers Safer, More Affordable Cancer Treatment
The treated mice displayed marked tumour regression and prolonged survival, demonstrating the therapeutic efficacy of the mRNA-LNP CAR-T cells. This innovative approach could revolutionise CAR-T ...
Johns Hopkins Medicine19d
Cystic Hygroma
What is a cystic hygroma? A cystic hygroma — or lymphangioma — is a birth defect that appears as a sac-like structure with a thin wall that most commonly occurs in the head and neck area of an infant.
Pharmabiz20d
Arcturus Therapeutics initiates patient dosing in phase 2 multiple ascending dose studies for CF and OTC deficiency
“ARCT-810 is the only mRNA therapy currently ... of OTC deficiency.” Cystic fibrosis is a life-shortening disease with a worldwide prevalence. Mutations in the cystic fibrosis transmembrane ...
Healthline20d
What to Know about Anger Management Therapy
Anger management therapy can include a host of different therapies such as cognitive behavioral therapy or psychodynamic therapy. Your therapist or psychologist will tailor any chosen therapy to ...
Business Wire22d
Arcturus Therapeutics Announces Initiation of Dosing in Phase 2 Multiple Ascending Dose Studies for Cystic Fibrosis (CF) and Ornithine Transcarbamylase (OTC) Deficiency
“ARCT-810 is the only mRNA therapy ... Fibrosis Cystic fibrosis is a life-shortening disease with a worldwide prevalence. Mutations in the cystic fibrosis transmembrane conductance regulator ...
Time26d
Scientists Are Racing to Develop a New Bird Flu Vaccine
What about an mRNA vaccine for H5N1? There isn't one yet, but several companies—including Moderna, Pfizer and GlaxoSmithKline (in collaboration with CureVac)—are working on such a shot.
FierceBiotech26d
Pfizer cuts losses on near-approval hemophilia gene therapy, adding to troubled Sangamo's woes
Pfizer gave Sangamo Therapeutics coal for Christmas, terminating a hemophilia A gene therapy pact to deprive the cash-strapped biotech of up to $220 million in milestones. Pfizer’s decision sent ...
Yahoo Finance26d
Why Moderna Inc. (MRNA) Is Skyrocketing
Shares of Moderna (NASDAQ:MRNA) closed the year on a strong note, rallying by 5.59 percent at $41.58 apiece as investors resorted to bargain-hunting after analysts regarded the company’s ...