Tag: Gene Therapy
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Curiosity Drives Discovery: How Michael Welsh’s Pursuit Shaped the Future of Cystic Fibrosis
Introduction: A career built on curiosity Curiosity has long been the spark that drives medical breakthroughs, and nowhere is that more evident than in the story of cystic fibrosis (CF). For over four decades, Dr. Michael J. Welsh, a renowned pulmonologist at University of Iowa Health Care, has turned questions into therapies, reshaping every aspect…
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Curiosity Drives Discovery: How CF Research Is Redefining the Future for Patients
Curiosity as the Catalyst for Cystic Fibrosis Breakthroughs Curiosity has long been the engine of medical progress, and nowhere is that more evident than in the field of cystic fibrosis (CF). For decades, researchers and clinicians have pursued answers to the disease’s stubborn questions, turning what seemed like intractable problems into actionable therapies. This ongoing…
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Gene Therapy Brings Hope for Child with Rare Parkinson’s
Hope Emerges from a Groundbreaking Gene Therapy In Singapore, a breakthrough in pediatric neuroscience is offering renewed hope to families affected by a rare Parkinson’s-like disease. A young patient, diagnosed soon after birth with a neurodegenerative condition characterized by stiffness, seizure-like episodes, and impaired motor and speech development, became the focus of an ambitious gene…
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Gene Therapy Brings Hope: Child with Rare Parkinson’s Disease Shows Remarkable Recovery
Hope Emerges from a Groundbreaking Gene Therapy In Singapore, a medical breakthrough has offered new optimism for families affected by a rare form of Parkinson’s disease in children. A successful gene therapy intervention has shown meaningful improvements in motor function, communication, and daily living activities for a young patient, sparking conversations about expanding access to…
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Groundbreaking Gene Therapy Brings Hope for Child with Rare Parkinson’s Disease
Hope for a Rare Parkinson’s Disease Patient In Singapore, a medical breakthrough has sparked cautious optimism for families affected by a rare form of Parkinson’s disease that strikes in childhood. A few weeks after a Spanish girl named Irai was born, doctors noticed severe motor and developmental challenges. She could not hold her head up,…
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Wiskott-Aldrich Gene Therapy: EU EMA Recommends Waskyra for WAS
What is Wiskott-Aldrich syndrome and the promise of gene therapy Wiskott-Aldrich syndrome (WAS) is a rare, inherited immune disorder caused by mutations in the WAS gene. The condition typically presents in infancy or early childhood with a mix of immune dysfunction, small blood cells (thrombocytopenia), eczema, and an increased risk of infections and autoimmune problems.…
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EU Considers First Gene Therapy for Wiskott-Aldrich Syndrome: Waskyra
EU Moves Toward First Gene Therapy for Wiskott-Aldrich Syndrome The European Medicines Agency (EMA) has recommended granting marketing authorization in the European Union for Waskyra (etuvetidigene autotemcel), a gene therapy designed to treat people aged 6 months and older with Wiskott-Aldrich syndrome (WAS) who carry mutations in the WAS gene. If approved, Waskyra would become…
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First EU Approval Consideration for WAS Gene Therapy: Waskyra (etuvetidigene autotemcel)
What is Waskyra and Who Could Benefit? The European Medicines Agency (EMA) has recommended granting marketing authorisation in the European Union for Waskyra (etuvetidigene autotemcel) to treat people aged 6 months and older with Wiskott-Aldrich syndrome (WAS) who carry a mutation in the WAS gene. Waskyra is a one-time, autologous gene therapy that aims to…
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Genezen and Humane Genomics Partner for Pediatric Liver Cancer
New Partnership to Accelerate Oncolytic Virus Therapy Genezen, a leading viral vector Contract Development and Manufacturing Organization (CDMO), and Humane Genomics, a biotechnology company focused on oncolytic viral therapies, announced a strategic manufacturing partnership to advance a new therapy for pediatric liver cancer. The collaboration combines Genezen’s manufacturing scale and regulatory compliance expertise with Humane…
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First-in-Human Trial: CTX310 Gene-Editing Therapy Halves Bad Cholesterol
Groundbreaking Step in Lipid Disorder Treatment In a landmark development for cardiovascular health, researchers in Australia have initiated a first-in-human trial of CTX310, a gene-editing therapy designed to dramatically reduce bad cholesterol (LDL) and triglycerides in individuals with difficult-to-treat lipid disorders. Early results suggest the therapy can halve levels of harmful lipids, offering a potential…