Tag #Genetic Diseases

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forbes.com
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CRISPR Therapy Shows Promise but Faces Rollout Challenges

The first CRISPR-based therapy, approved in late 2023, offers a cure for previously untreatable diseases; however, slow rollout due to manufacturing and accreditation challenges highlights the need for regulatory adaptations and technological advancements in CRISPR enzyme delivery.

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lexpansion.lexpress.fr
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Pre-conception Genetic Testing: Potential to Reduce Severe Genetic Diseases in France

In France, 15,000-20,000 new cases of severe genetic diseases are diagnosed yearly, impacting 2-3% of the population. An Australian study showed pre-conception genetic testing identified 2% of 9,100 couples at risk, with 75% using this information for prenatal or preimplantation diagnosis, suggestin...

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elpais.com
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Diagnóstico genético identifica el origen de enfermedades neuromusculares en niños

Una nueva plataforma de diagnóstico genético ha identificado la causa de enfermedades neuromusculares en 23 niños, utilizando secuenciación del genoma en trío y del transcriptoma, logrando una tasa de éxito del 40% en un estudio de 58 pacientes pediátricos.

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pda.stav.kp.ru
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Russian Boy with Duchenne Muscular Dystrophy Needs $3 Million for Gene Therapy

Vladislav Paradov, a Russian boy, was diagnosed with Duchenne muscular dystrophy (DMD) at age three, a condition that until recently had no cure. His family is now fundraising $3 million for a new gene therapy treatment available in Dubai.

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votreargent.lexpress.fr
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Genetic Testing Could Prevent Thousands of Severe Genetic Disease Births in France

In France, 15,000–20,000 new cases of severe genetic diseases are diagnosed annually; Australia's genetic testing program, identifying ~2% of couples at risk, resulted in 3/4 using this information for preventative measures, suggesting a potential for reducing severe genetic disease births in France...

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Showing 1 to 5 of 5 results