Woman with Rare Facial Condition Chooses Self-Acceptance
Laxmi Chaudhari faced rejection and underwent surgeries trying to fix her rare facial condition. Then she made a harder choice: accepting herself exactly as she is.
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12132 results for "rare conditions"
Tennis Star Defies Rare Condition to Reach Career High
British tennis player Francesca Jones, who was told she'd never play professionally due to a rare genetic condition, achieved direct entry into the Australian Open main draw for the first time after a career-changing 2024 season. The 25-year-old went from considering retirement to climbing back inside the world's top 75.
New Drug Cuts Rare Disease Triglycerides by 32%
UK regulators just approved olezarsen, the first treatment in years for familial chylomicronemia syndrome, a rare genetic condition that can cause life-threatening pancreatic inflammation. Clinical trials showed patients reduced dangerous fat levels by a third while experiencing fewer emergency attacks.
Scientists Find Cure for Gut Condition That Makes You Drunk
Researchers at Mass General Brigham have discovered how to treat auto-brewery syndrome, a rare condition where gut bacteria turn food into alcohol and make people drunk without drinking. The breakthrough could help patients who've struggled for years with unexpected intoxication.
Gene Cream Could Cure Rare Skin Disease With One Treatment
Scientists at the University of British Columbia have created the first topical gene therapy that corrects faulty DNA directly in human skin, offering hope for patients with painful genetic skin disorders who currently have no cure. The breakthrough treatment could eventually help millions with conditions ranging from rare inherited diseases to common eczema.
Europe Rewrites Rules to Fast-Track Rare Disease Cures
After a decade of stagnation where only 5% of rare diseases had treatments, Europe is overhauling outdated regulations to bring hope to 30 million patients. New pathways are finally treating ultra-rare conditions differently than common illnesses.
Indonesian Hospital Shows How to Track Rare Diseases
A hospital in Indonesia built a successful rare disease registry in just two years, offering hope for similar efforts in Malaysia. The achievement could help 45 million people across Southeast Asia living with rare conditions get better care. #
Austin Lab Creates Custom Drug to Save Baby With Rare Disease
Scientists in Austin are developing a one-of-one therapy for baby Everett Blomstrom, who has an ultra-rare genetic disease affecting fewer than 50 people worldwide. The experimental treatment could be ready in two years and represents a breakthrough in personalized medicine for rare conditions.
AstraZeneca Drug Shows Promise for Rare Kidney Disease
A breakthrough treatment for a devastating rare kidney condition just moved closer to reality. AstraZeneca's Ultomiris has shown encouraging results in clinical trials, offering new hope to patients with limited options.
New Drug Doubles Growth Rate for Kids with Rare Disorders
Children with rare genetic growth disorders grew twice as fast after starting a groundbreaking treatment, offering hope where few options existed before. The therapy worked especially well for kids whose condition directly matched the drug's target.
Shanghai Team Cracks Code on Rare Facial Disorder
A Shanghai hospital has unlocked the mystery behind a devastating facial condition and created the first targeted treatment, bringing hope to patients worldwide. The breakthrough includes the world's first patient database for the rare disorder.
FDA Proposes Fast-Track for Rare Disease Gene Therapies
The FDA just announced a new pathway that could bring life-saving gene editing treatments to patients with rare diseases in record time. For families battling conditions once considered untreatable, this could change everything.
Boca Raton AI Breakthrough Cuts Rare Disease Wait Time
A family's agonizing years-long wait for their child's rare disease diagnosis has sparked an artificial intelligence breakthrough in Boca Raton that could help thousands of other families get answers faster. The innovative system is already showing promise in identifying conditions that typically take years to diagnose.
FDA Approves First Treatment for Rare Menkes Disease
The FDA just approved the first treatment for Menkes disease, a rare genetic disorder that has claimed countless young lives. Copper histidinate could help babies with this devastating condition finally have a fighting chance.
UK Approves New Treatment for Rare Swelling Disorder
People with hereditary angioedema, a rare genetic condition causing painful and sometimes life-threatening swelling, now have a new prevention option in the UK. The treatment could help thousands reduce attacks and reclaim their daily lives.
Hidden Gene Solves Mystery of Rare Movement Disorder
Scientists analyzed nearly 3,000 patients and discovered that a gene previously thought to only affect immunity actually causes a rare neurological condition. The breakthrough could help diagnose people who've lived years without answers about their movement difficulties.
UC Davis Unlocks Mystery of Rare Childhood Brain Diseases
Scientists have mapped how a tiny cellular machine malfunctions in rare childhood brain disorders, offering the first clear path toward future treatments. The discovery could help diagnose dozens of neurological conditions that currently leave families without answers.
New Drug Slashes Fat Levels for Rare Genetic Disease
People with a rare genetic disorder that causes dangerous fat buildup in their blood now have a breakthrough treatment that cuts triglyceride levels by nearly a third. The UK just approved olezarsen for adults with familial chylomicronemia syndrome, a condition that can trigger life-threatening pancreas inflammation.
AI Cuts Rare Disease Drug Research From 100 Days to 17 Hours
A nonprofit is using AI to match 4,000 existing drugs with 18,000 diseases in less than a day, potentially bringing treatments to millions suffering from rare conditions. Every Cure has already identified 10 promising treatments since 2022.
Céline Dion Returns to Stage After Beating Rare Syndrome
Céline Dion is performing again after a years-long battle with stiff person syndrome, a rare neurological condition that nearly ended her legendary career. The superstar announced 10 comeback shows in Paris starting this September.
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