Independent.ie3d
Wicklow mother calls for access to life-saving therapy for son with rare form of Cystic Fibrosis
His rare gene means he is currently excluded from modulator therapy Kaftrio ... are being supported by Cystic Fibrosis Ireland, and believes the only solution for people with CF like Aaron ...
pharmaphorum2d
Boehringer joins UK academics in battle against cystic fibrosis
Boehringer Ingelheim (BI) and the UK Cystic Fibrosis Gene Therapy Consortium announced a global collaboration to develop a first-in-class, long-term therapy for patients with cystic fibrosis (CF).
imt.ie6d
Call for children with cystic fibrosis and rare genes to gain access to life-saving therapy
Some people with cystic fibrosis still do not have access to the life ... Louise Patterson’s son, Aaron, has a rare gene, which means he is excluded from this therapy. “Currently in Ireland, only gene ...
Independent.ie3d
‘Time is precious’ – children with rare cystic fibrosis gene denied access to potentially life-extending therapies
Children whose cystic fibrosis is caused by a rare gene variant are excluded from a new treatment which could potentially slow the decline in their lung function, it has emerged. A group of ...
Healio6d
Vanzacaftor, tezacaftor, deutivacaftor safe in children with cystic fibrosis
Patients aged 6 to 11 years with cystic fibrosis receiving 24-week once-daily vanzacaftor/tezacaftor/deutivacaftor generally ...
Forbes5d
Genetic Swiss Army Knife: New Tool For Gene Editing And Therapy
The implications of mvGPT for gene therapy are significant. The ability to execute multiple genetic edits simultaneously opens new avenues for treating complex conditions. Recent research ...
Sky News Australia2d
'I have that guilt': I'm A Celeb star Reggie Bird opens up about her son's battle with the genetic disorder cystic fibrosis
Sadly, Reggie’s son was diagnosed with the genetic disorder cystic fibrosis at four weeks old, and now requires daily medication to manage symptoms. The disease impairs the normal clearance of ...
Medical Xpress6d
Personalized therapy for rare genetic diseases: Patient-derived organoids offer new hope
The study, titled "Rapid and scalable personalized ASO screening in patient-derived organoids," was published in the journal Nature.Many labs already generate patient-derived induced pluripotent ...
Nature1d
Gene therapy articles from across Nature Portfolio
An in vivo base editing approach targeting the PRNP gene led to a 52% increase in the lifespan of mouse models inoculated with the most common sporadic and genetic types of human pathogenic prion ...
Cystic Fibrosis Foundation3d
About Cystic Fibrosis
Learn about cystic fibrosis, a genetic disorder that affects the lungs, pancreas, and other organs, and how to treat and live with this chronic disease. 12 min read What Is Cystic Fibrosis? Cystic ...
The Telegraph5d
Jadavpur University’s TalkClubb Organises Psychoanalytic Therapy Workshop
Dr. Suchismita Sinha, a faculty member at Sarojini Naidu College for Women, offered a contemporary perspective on psychoanalytic therapy, emphasizing its relevance in addressing modern-day mental ...