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69 results for "rare disease"
Children across Europe with a painful swallowing disorder finally have a medicine made just for them. The EU just approved Jorveza oral suspension, ending years of kids taking adult medications not designed for their bodies.
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.
Jeff Allen won $10 million on "Beast Games" and is using his prize to fund research for his son Lucas's rare genetic disorder. His efforts could unlock treatments for millions with Alzheimer's and Parkinson's.
When Julia Friar's newborn son was diagnosed with a life-threatening genetic disorder, she transformed her fear into action. Now she's helping families across the country while fundraising for a cure.
Morocco just launched a Precision Medicine Hub that will help thousands of patients with rare diseases get faster diagnoses and coordinated care. The new national center brings together advanced genetic testing, specialist teams, and patient support under one roof.
A new medication just became the first therapy to directly target the damaged cells causing a serious kidney disease with no approved treatments. The breakthrough offers real hope for patients with FSGS, a rare condition that often leads to kidney failure.
When two Tennessee mothers learned their children share an ultra-rare neurological disorder, they discovered something remarkable: a cure exists. Now they're hosting a superhero run to help bring that gene therapy to their kids and fewer than 500 other patients worldwide.
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.
A Philadelphia nonprofit founded by a doctor who survived a rare disease just landed $76 million to help millions of patients find treatments hiding in plain sight. Every Cure's AI tool searches thousands of existing drugs to find new uses for rare diseases that lack treatments.
A 19-year-old from British Columbia became the first person ever cured using "prime editing," a breakthrough gene therapy that fixed a fatal immune disorder. The treatment freed him from daily medication and the constant fear of life-threatening infections.
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.
Families battling ultra-rare diseases just got a major breakthrough: the FDA announced a new pathway that will speed life-saving treatments to patients who've been told for decades "there aren't enough of you to study." A baby boy's miraculous CRISPR therapy inspired the change.
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.
The FDA just unveiled a new approval process that could bring life-saving treatments to millions of Americans with rare diseases in months instead of years. Families who've heard "wait for the science to catch up" finally have reason to hope.
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 newly approved medication could help people with Prader-Willi syndrome control the constant, overwhelming hunger that defines their daily lives. One mother shares why she's hopeful this treatment will give her son Robert a chance at greater independence and happiness.
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.
Scientists used a precise gene-editing tool to fix a rare brain disorder in mice, reversing learning problems and behavioral issues after birth. The breakthrough suggests that genetic brain damage might not be permanent, offering new hope for treating neurodevelopmental conditions.
The U.S. just approved the largest federal ALS research budget in history at $315 million. New treatments targeting the disease's root causes are showing real promise for the first time.
A 60-year-old woman who kept losing consciousness finally got answers when doctors found a tiny tumor in her pancreas and removed it using robotic surgery. Her blood sugar levels normalized immediately after the procedure.
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