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18 results for "genetic diseases"
After 25 years of research, scientists have submitted the first application to treat genetic diseases in fetuses before birth. The FDA told them they can skip animal testing because the therapy is already proven safe.
Scientists matched vitamins to genetic diseases and discovered that vitamin B3 dramatically extends survival in mice with NAXD deficiency, a disorder that usually kills children within months. This genetics-first approach could unlock safe, affordable treatments for dozens of rare genetic diseases.
Latus Bio just secured $42 million to advance treatments for two devastating genetic diseases, including Huntington's. The funding brings hope to families waiting for breakthroughs in conditions that currently have no cure.
A decades-old medicine originally used for African sleeping sickness is giving new hope to families battling Bachmann-Bupp syndrome, one of the world's rarest genetic diseases. Just 20 people worldwide have been diagnosed, but five patients are already showing visible improvements.
A one-time gene-editing treatment just became the first to succeed in a major clinical trial, reducing dangerous swelling attacks by 87% for people with a rare genetic condition. The breakthrough could change what's possible for treating genetic diseases.
A new FDA pathway could cut the time to get personalized CRISPR treatments from four years to just three months, making life-saving gene editing accessible to thousands of children born with rare genetic diseases. Baby KJ Muldoon became the first person to receive this revolutionary treatment in February 2025.
A Boca Raton company turned one father's four-year search for answers into an AI tool that diagnoses rare genetic diseases in 10 seconds instead of years. GENA has already helped diagnose 160,000 cases and could save countless children from the same waiting game.
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.
The FDA just unveiled a breakthrough framework that could bring life-saving gene therapies to thousands of children with rare diseases in a fraction of the time. For families who've watched their kids suffer while treatments stall, this changes everything.
Ty Sperle, 18, became the first person in the world cured of chronic granulomatous disease through groundbreaking gene editing treatment. His success opens the door for curing countless other rare genetic diseases affecting children everywhere.
Scientists have created a precise gene-editing tool that could offer permanent treatment for cystic fibrosis patients, potentially replacing daily medications that cost hundreds of thousands of dollars per year. The breakthrough could transform care for people living with this and other genetic diseases.
A 9½-month-old baby became the first person ever to receive a custom gene-editing treatment designed specifically for his unique mutation. The breakthrough offers hope for thousands of rare genetic diseases that were once considered unreachable.
Scientists have developed a new gene editing approach that spreads healing changes from cell to cell, dramatically increasing its reach in the body. This breakthrough could transform treatment for countless genetic diseases that were previously too difficult to address.
Google DeepMind just opened up its DNA-reading AI to help scientists worldwide crack the code of genetic diseases. Nearly 3,000 researchers are already using it to fight cancer, brain disorders, and infections.
Google's DeepMind just unveiled an AI that can read and predict the impact of mutations in the 98% of human DNA scientists barely understand. This breakthrough could accelerate the discovery of treatments for everything from rare genetic diseases to cancer.
Australian researchers developed an AI system that makes gene editing safer by protecting healthy genes during treatment. The breakthrough could transform how doctors treat genetic diseases like cystic fibrosis.
Melbourne researchers developed an AI tool that creates molecular "off switches" for CRISPR gene editing in just 8 weeks. The breakthrough could help make gene therapy treatments safer and more accessible for patients with genetic diseases.
Scientists just proved that two harmful genetic mutations can actually restore normal function when combined, confirming a 60-year-old hypothesis. This breakthrough could transform how doctors diagnose and treat thousands of rare genetic diseases.