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8097 results for "disease-modifying therapy"
A groundbreaking RNA therapy just succeeded in early trials for a rare muscular dystrophy affecting thousands, and it could become the first treatment that actually modifies the disease's progression. Novartis's del-brax showed clear signs of reducing muscle damage in patients with facioscapulohumeral muscular dystrophy.
For the first time, a gene therapy has shown dramatic results in slowing Huntington's disease, offering hope to 8,000 UK families who've had no treatment options until now. Patients receiving the higher dose experienced 75% less disease progression over three years.
A groundbreaking gene therapy called AMT-130 has slowed Huntington's disease progression by 75% in clinical trials, offering hope to families who've waited 154 years for effective treatment. The one-time treatment could preserve patients' ability to walk, speak, and connect with loved ones for years longer.
The FDA reversed course on a groundbreaking gene therapy for Huntington's disease, bringing new hope to thousands of families. The treatment from biotech company UniQure can now move forward toward U.S. approval.
A 41-year-old mother who spent her life battling sickle cell disease just became the first adult in Maryland to receive a potentially curative gene therapy using her own cells. After decades of hospital stays and missing family moments, Jessica Ceja now has hope for a life without the painful disease.
Children and adults with a rare genetic disease that required eating every few hours can now receive a one-time gene therapy treatment. The FDA approval marks the first treatment to address the root cause of glycogen storage disease type Ia.
A groundbreaking cell therapy has successfully treated stiff person syndrome, a rare neurological disorder that causes severe muscle stiffness and mobility problems. If approved by the FDA this summer, it would become the first treatment for the condition and the first personalized CAR-T therapy for any autoimmune disease.
A groundbreaking treatment for myotonic dystrophy type 1 reduced toxic proteins by 40% and improved muscle function in patients who've never had disease-modifying options. The New England Journal of Medicine just published results that could lead to the first approved therapy for this progressive, often fatal disease.
The FDA just made it easier for life-saving gene therapies to reach patients with ultra-rare diseases who previously had few treatment options. The new pathway means families won't have to wait years for cures when the science shows real promise.
A breakthrough treatment originally designed for cancer is giving hope to over 5 million Indians living with autoimmune diseases. CAR T-cell therapy is helping patients with lupus and arthritis reduce lifelong medications by resetting their faulty immune systems.
A pharmaceutical company has brought new hope to families affected by a rare disease by resurrecting a gene therapy program that was once thought lost. The treatment offers a second chance for patients in a community that had faced devastating setbacks.
A world-first inhaled RNA therapy that targets the root cause of lung disease has entered human trials in Australia. The breakthrough could transform treatment for nine million Australians living with chronic lung conditions.
University of Toronto researchers engineered a breakthrough RNA treatment that could tackle thousands of genetic diseases with a single approach. The therapy teaches cells to ignore faulty "stop signals" that prevent life-saving proteins from forming.
A one-time brain treatment that could slow Huntington's disease progression by 75% is now under review by US and UK regulators. If approved, it would be the first therapy to directly silence the gene driving this devastating neurological disorder.
A Louisiana man just became the first person in the state to be functionally cured of sickle cell disease using advanced gene therapy. The breakthrough offers hope to thousands living with the painful genetic blood disorder.
A breakthrough cancer treatment is giving hope to millions living with autoimmune diseases. Early trials show CAR T-cell therapy could offer a one-time "immune reset" instead of lifelong medications.
Millions living with lupus, rheumatoid arthritis, and multiple sclerosis could soon trade a lifetime of medication for a single treatment. Sail Biomedicines is unveiling breakthrough data on a therapy that reprograms immune cells inside the body to potentially cure autoimmune diseases.
A 21-year-old bioengineering student diagnosed with a rare muscle disease at 13 is now engineering a potential cure for herself and nearly 1 million others worldwide. Héloïse Hoffmann just won a $100,000 fellowship to advance her groundbreaking gene therapy research.
Two Philadelphia research centers just received millions in federal funding to develop gene therapies that could cure rare diseases within three years. The ambitious timeline could bring hope to families who've been waiting decades for treatments.
The FDA just made it easier for millions of Americans with rare diseases to access life-saving gene therapies without waiting years for traditional clinical trials. Families who were told to "wait for science to catch up" now have real hope.
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