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Infant with rare, incurable disease is first to successfully receive personalized gene therapy treatment

Science Daily: Pharmacology News

A research team has developed and safely delivered a personalized gene editing therapy to treat an infant with a life-threatening, incurable genetic disease. The infant, who was diagnosed with the rare condition carbamoyl phosphate synthetase 1 (CPS1) deficiency shortly after birth, has responded positively to the treatment.

Treatment 321
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Novel stem cell therapy repairs 'irreversible' corneal damage in clinical trial

Science Daily: Pharmacology News

Results from a phase 1/2 clinical trial of a novel stem cell treatment for cornea injuries found 14 patients treated and tracked for 18 months had a more than 90% success rate at restoring the cornea's surface and improvements in vision.

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New chronic pain therapy retrains the brain to process emotions

Science Daily: Pharmacology News

Researchers have created an effective therapy for chronic pain that reduces pain intensity by focusing on emotional regulation.

Therapies 256
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100x improvement in sight seen after gene therapy trial

Science Daily: Pharmacology News

The vision of people with a rare inherited condition that causes them to lose much of their sight early in childhood was 100 times better after they received gene therapy to address the genetic mutation causing it.

Therapies 330
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Gene therapy gets a turbo boost

Science Daily: Pharmacology News

Gene therapy, the idea of fixing faulty genes with healthy ones, has held immense promise. For decades, scientists have dreamt of a future where genetic diseases, such as the blood clotting disorder hemophilia, could be a thing of the past. But a major hurdle has been finding a safe and efficient way to deliver those genes.

Therapies 312
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New gene therapy for muscular dystrophy offers hope

Science Daily: Pharmacology News

A new gene therapy treatment for Duchenne muscular dystrophy (DMD) shows promise of not only arresting the decline of the muscles of those affected by this inherited genetic disease, but perhaps, in the future, repairing those muscles.

Therapies 310
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First therapy chatbot trial shows AI can provide 'gold-standard' care

Science Daily: Pharmacology News

Researchers conducted the first clinical trial of an AI-powered therapy chatbot and found that, on average, people with diagnosed mental disorders experienced clinically significant improvements in their symptoms over eight weeks, according to new results.

Therapies 315