Congenital absence of the vas deferens

SpliSense Presents Preclinical Data for SPL23-ASO, mRNA-Altering Drug Candidate for the Treatment of Cystic Fibrosis, at the European Cystic Fibrosis Conference

Retrieved on: 
Friday, June 11, 2021
Cystic fibrosis, Medical specialties, Branches of biology, Cystic fibrosis transmembrane conductance regulator, Clinical medicine, Draft:Cystic Fibrosis: An Overview, Congenital absence of the vas deferens

symposium at the 44th European Cystic Fibrosis Digital Conference , June 9-12, 2021.

Key Points: 
  • symposium at the 44th European Cystic Fibrosis Digital Conference , June 9-12, 2021.
  • SpliSense utilizes short, precisely targeted proprietary RNA stretches called ASOs to correct various mutations in the CFTR mRNA.
  • "We are pleased to add another promising preclinical candidate to our pipeline," said Gili Hart, PhD, CEO, SpliSense.
  • Cystic fibrosis (CF) is a genetic disease that leads to respiratory infections and disabilities and affects over 90,000 people worldwide.

Survey Finds People with Cystic Fibrosis Using a CFTR Modulator Have Positive View of Treatment Plans, HCP Relationships, Future

Retrieved on: 
Tuesday, November 26, 2019
Cystic fibrosis, Branches of biology, Medical specialties, RTT, Membrane proteins, Channelopathies, Congenital absence of the vas deferens, Ivacaftor

The survey illuminates the perspectives and experiences of people impacted by cystic fibrosis.

Key Points: 
  • The survey illuminates the perspectives and experiences of people impacted by cystic fibrosis.
  • CFTR modulators are only indicated for use with certain mutations and, depending on the specific treatment, only indicated for certain age groups.
  • Additionally, patient respondents who are currently using a CFTR modulator appear to have more positive relationships with their healthcare professionals.
  • Patients who have never used a CFTR modulator have a less optimistic view of how CF affects both their present and their future.

Vertex Receives Approval for SYMDEKO® (tezacaftor/ivacaftor and ivacaftor) in Australia, to Treat the Underlying Cause of Cystic Fibrosis in People aged 12 and Older with Certain CFTR Gene Mutations

Retrieved on: 
Tuesday, March 12, 2019
Health, Biotechnology, Pharmaceutical, Medical Supplies, Cystic fibrosis, Branches of biology, Chemical compounds, Medical specialties, Cystic fibrosis transmembrane conductance regulator, Tezacaftor, Transepithelial potential difference, Congenital absence of the vas deferens

SYMDEKO will be considered for Australian reimbursement for eligible CF patients aged 12 years and older at the March meeting of the Pharmaceutical Benefits Advisory Committee.

Key Points: 
  • SYMDEKO will be considered for Australian reimbursement for eligible CF patients aged 12 years and older at the March meeting of the Pharmaceutical Benefits Advisory Committee.
  • We are delighted that the Therapeutic Goods Administration in Australia recognized the safety profile and efficacy of SYMDEKO.
  • Cystic Fibrosis (CF) is a rare, life-shortening genetic disease affecting approximately 75,000 people in North America, Europe and Australia.
  • CF is caused by a defective or missing cystic fibrosis transmembrane conductance regulator (CFTR) protein resulting from mutations in the CFTR gene.