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93 results for "gene therapy"
A mother is running the London Marathon to fund research for gene therapies that could save her daughter from kidney failure. Her eight-year-old was diagnosed with a rare, incurable form of nephrotic syndrome that currently has no treatment options.
Pharmaceutical company Roche announced a new clinical trial that could bring a life-changing gene therapy for Duchenne muscular dystrophy to young patients across Europe. The move comes after extensive feedback from families and regulators who want to see this treatment option become available.
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 breakthrough gene therapy wiped out aggressive brain tumors in 83% of cases with a single dose and no side effects for nearly a year. The treatment also taught the immune system to prevent the cancer from returning.
Researchers at Harvard Medical School have developed a breakthrough gene-editing method that successfully silences the extra chromosome causing Down syndrome in up to 40% of lab cells. While still early stage, this proof-of-concept study opens the door to the first genuine treatment for one of the most common genetic conditions.
A new gene therapy has reduced seizures by up to 85% in children with Dravet syndrome, a devastating form of epilepsy that begins in infancy. Kids in the trial are not only having fewer seizures but are making friends and enjoying life alongside their peers for the first time.
Five people with beta thalassemia no longer need regular blood transfusions after receiving a groundbreaking gene therapy treatment developed in China. Their recovery marks a major milestone in making cutting-edge genetic medicine accessible worldwide.
A new partnership between Canadian and Japanese biotech companies could slash the cost of life-saving gene therapies by making them easier to produce. The collaboration aims to help more patients access treatments that were once financially out of reach.
Chinese researchers just proved that a safer, more precise gene editing tool can cure β-Thalassaemia, a life-threatening blood disorder affecting millions worldwide. Unlike earlier methods, this new system made zero mistakes while editing patient cells.
Scientists have developed a gene therapy that completely eliminated aggressive brain tumors in 83% of animal trials, with no recurrence over 11 months. The breakthrough treatment for glioblastoma, the deadliest brain cancer, uses a "Trojan horse" approach that kills cancer cells while training the immune system to fight back.
A breakthrough gene editing treatment has achieved what doctors call a "functional cure" for sickle cell disease, with 27 out of 28 patients experiencing zero painful crises after receiving the therapy. The one-time treatment edits patients' own blood cells to correct the genetic mutation, offering new hope for 100,000 Americans living with this painful disease.
When doctors couldn't help her son's rare brain disorder, Amber Freed learned genetics, coordinated researchers worldwide, and raised millions to create the first gene therapy for his condition. Maxwell, now 8, became the first person ever to receive the groundbreaking treatment.
A mother who raised $1.3 million to fund a controversial treatment for her son's fatal muscle disease just saw clinical trial results that stunned experts. The experimental drug uses a counterintuitive approach: fixing broken genes by breaking them a little more.
Ten people born deaf can now hear after a single gene therapy injection, with some improvements appearing in just one month. Children showed the most dramatic results, including one seven-year-old who began having everyday conversations with her mom.
A single gene-editing treatment has freed nearly all trial patients from the crushing pain crises that define sickle cell disease. Doctors are calling it a functional cure that uses patients' own cells to rewrite their future.
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.
Twenty-seven out of 28 patients with severe sickle cell disease are now living pain-free after a groundbreaking gene editing treatment. The one-time therapy is offering new hope to people living with a genetic disorder that typically shortens life expectancy to the mid-40s.
Two sisters born with a fatal genetic disorder are thriving thanks to breakthrough treatments that didn't exist a decade ago. Their family's story shows how medical innovation is transforming once-hopeless diagnoses into lives full of possibility.
The only multidisciplinary ALS clinic in the Mountain West now offers groundbreaking gene therapy for patients with genetic forms of the disease. University of Utah Health's team brings together 10+ specialists in one visit, making life easier for the 30,000 Americans living with ALS.
Scientists have developed the first brain-targeted gene therapy that stops chronic pain without addiction risk, offering hope to 50 million Americans. Early tests show it works like morphine but skips the dangerous side effects that fuel the opioid crisis.
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