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6007 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.
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 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.
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.
A breakthrough cell therapy cut the death rate in half for patients with advanced liver disease, offering hope beyond transplants. After four years, 70% of patients treated were alive without needing a transplant, compared to just 40% who received standard care.
Researchers at USC are using AI to fast-track lifesaving cell and gene therapies for children with rare diseases, transforming how treatments reach the smallest patient populations. The new framework could help families access cutting-edge cures months or even years sooner.
Families with rare diseases who once had to travel overseas and raise hundreds of thousands of dollars for life-saving treatments now have hope. Congress just introduced legislation that would let patients access personalized gene therapies without waiting for FDA approval.
The FDA just approved testing of a revolutionary therapy that could cure lupus, rheumatoid arthritis, and other autoimmune diseases without harsh chemotherapy. The breakthrough could help 15 million Americans escape lifelong medications.
After decades of research, gene therapy is bringing lasting relief to sickle cell patients after just one treatment. UCLA researchers are leading the charge to cure a disease that affects 100,000 Americans.
A German woman who spent weeks bedridden with three life-threatening autoimmune diseases is now symptom-free after a single treatment with engineered immune cells. She hasn't needed medication since receiving the therapy that doctors called her "last chance."
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