#gene-therapy

#gene-therapy

Insurers' refusal to cover innovative gene therapies for conditions like sickle cell disease jeopardizes patient access, necessitating creative financing solutions.

Commercially approved gene therapy offers hope to sickle cell patients like Kendric Cromer, potentially leading to a cure.

Gene therapy shows promise in protecting vision by improving retinal cell function in glaucoma patients.

New research reveals potential therapies for retinal damage using gene therapy to combat Prox-1 protein's inhibitory effect on retinal regeneration.

CRISPR-based gene therapy improved vision in individuals with inherited blindness.

Colton Belluzzo received groundbreaking gene therapy for Duchenne muscular dystrophy, showing promise in slowing disease progression.

A patient died from acute liver injury while using Sarepta's Elevidys gene therapy for muscular dystrophy, causing a major stock market reaction.

The Kasner family faces Duchenne muscular dystrophy with resilience and a strong support network, embracing love and life despite the challenges.

2025 is poised to be a transformative year for neuroscience with breakthroughs in brain mapping and AI-led therapies.

Casgevy gene therapy approved for severe thalassaemia on NHS in England, potentially offering a lifetime cure with significant benefits for patients.

Successful gene therapy trial for genetic deafness promising for future availability.

PRGN-2012 shows promise as a gene therapy for recurrent respiratory papillomatosis, potentially reducing the need for multiple surgeries.

Tech innovations in October highlighted significant funding rounds for companies like Knave, Purespring, and Basecamp Research.

uniQure plans to raise $75M through a public offering amid promising progress in gene therapies for Huntington's disease.

2023 highlights include breakthroughs in gene therapy for sickle-cell anemia and a new HIV prevention drug that shows remarkable efficacy.

Sam Berns' life epitomizes the urgent need for advancements in genetic treatments, particularly for disorders like progeria that code for rapid aging.

Gene therapy trial restores hearing in deaf children, showing promise for future treatments.

Significant milestone in gene therapy for treating deafness.