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2782 results for "** gene therapy"
Children born completely deaf are hearing whispers for the first time thanks to an experimental gene therapy that has shown remarkable success in the largest study of its kind. The treatment could become the first FDA-approved gene therapy for deafness within months.
A safer gene therapy for Duchenne muscular dystrophy just hit a major milestone, bringing hope to families facing this fatal muscle disease. The treatment could offer a better option than current therapies linked to serious side effects.
A gene therapy called ELEVIDYS is helping young boys with Duchenne muscular dystrophy maintain their ability to walk, run, and stand years after a single treatment. Three years after receiving the therapy, treated children showed 70% less decline in muscle function compared to untreated children.
A nonprofit and patient foundation just committed $7.65 million to develop a gene therapy for a rare muscular dystrophy with no current treatments. The partnership will take the therapy from initial design all the way to human clinical trials.
A groundbreaking gene therapy trial has achieved what doctors call a "functional cure" for sickle cell disease, eliminating painful crises in 27 out of 28 patients. The one-time treatment uses gene editing to fix the blood disorder that has killed patients decades too soon.
Serenity Cole, 18, spent years in constant pain from sickle cell disease. Now she's pain-free thanks to a revolutionary payment deal where drug companies only get paid if their $2-3 million gene therapies actually work.
A groundbreaking partnership between Canadian and Japanese biotech companies could slash the cost of life-saving gene therapies. With government support, they're creating technology to produce treatments faster and cheaper than ever before. --- ##
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 groundbreaking gene therapy trial in China gave hearing to 90% of deaf children who received it, with some able to hear whispers. The treatment even worked in adults, including a 32-year-old, marking a major breakthrough three years after the first injections.
Children born deaf are now hearing their mother's voice for the first time, thanks to a groundbreaking gene therapy that restores natural hearing. The FDA just approved the treatment, and the company is offering it completely free across the United States.
After 20 years of groundbreaking brain research, a powerhouse science team is pivoting to develop gene therapies for Alzheimer's and Parkinson's. Their expertise could unlock new treatments for millions living with brain diseases.
Scientists who created gene therapies that cure inherited blindness and blood disorders just won six $3 million prizes. Their discoveries are transforming devastating diseases from hopeless diagnoses into treatable conditions.
A groundbreaking gene therapy trial has restored hearing in 90% of people born profoundly deaf, with improvements lasting over two years. The treatment worked across ages from infants to adults, offering new hope beyond cochlear implants.
Scientists at UC San Diego have developed a gene therapy that shields brain cells from toxic proteins linked to dementia, successfully preserving memory in mice. The treatment could offer hope for millions facing Alzheimer's, frontotemporal dementia, and ALS.
Researchers who developed the first FDA-approved gene therapy for inherited blindness are among this year's $3 million Breakthrough Prize winners. Their work is opening doors for treatments that were once considered impossible.
Children born deaf are now hearing and speaking for the first time, thanks to a breakthrough gene therapy that reversed a rare inherited form of deafness. The treatment's effects have lasted up to 2.5 years, the longest follow-up yet reported.
The FDA just approved the first gene therapy to treat genetic hearing loss, giving hope to dozens of families each year whose babies are born deaf. Even better? The company is making it free for all U.S. patients.
A 26-year-old Louisiana man received the state's first gene therapy treatment for severe hemophilia A, potentially ending his need for lifelong infusions. The breakthrough treatment allows his body to produce the clotting protein it previously couldn't make.
A Kansas City hospital will multiply its capacity to deliver breakthrough cell and gene therapies that can cure diseases with a single treatment. The expansion promises to bring life-changing care closer to home for families across the Midwest.
Children born with a specific type of genetic hearing loss can now hear their parents' voices and dance to music, thanks to the FDA's approval of the first-ever gene therapy for inherited deafness. The one-time treatment helps patients avoid cochlear implants and restores natural hearing.
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