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13274 results for "rare disease therapy"
Children and adults with a rare genetic disease that required eating every few hours can now receive a one-time gene therapy treatment. The FDA approval marks the first treatment to address the root cause of glycogen storage disease type Ia.
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.
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.
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.
Three major research institutions joined forces to create a nonprofit that will develop affordable gene therapies for rare diseases and share their methods with doctors worldwide. The initiative could transform personalized treatments from million-dollar fundraising campaigns into standard medical procedures.
Two Philadelphia research centers just received millions in federal funding to develop gene therapies that could cure rare diseases within three years. The ambitious timeline could bring hope to families who've been waiting decades for treatments.
A groundbreaking RNA therapy just succeeded in early trials for a rare muscular dystrophy affecting thousands, and it could become the first treatment that actually modifies the disease's progression. Novartis's del-brax showed clear signs of reducing muscle damage in patients with facioscapulohumeral muscular dystrophy.
Researchers at USC are using AI to fast-track lifesaving cell and gene therapies for children with rare diseases, transforming how treatments reach the smallest patient populations. The new framework could help families access cutting-edge cures months or even years sooner.
Families with rare diseases who once had to travel overseas and raise hundreds of thousands of dollars for life-saving treatments now have hope. Congress just introduced legislation that would let patients access personalized gene therapies without waiting for FDA approval.
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.
The FDA just created a faster pathway for patients with rare genetic disorders to access personalized gene therapies without lengthy clinical trials. While some experts celebrate this breakthrough for desperately ill patients, others urge caution about adequate safety monitoring.
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.
A federal health agency just launched a $160 million program to develop personalized gene editing treatments for rare diseases, with clinical trials expected to start within three years. Seven research teams are racing to bring hope to patients who've long waited for cures.
A one-time gene therapy targeting a devastating inherited heart condition just earned special FDA status, bringing hope to families facing early heart failure. The treatment addresses the root genetic cause rather than just managing symptoms.
Children and adults with a rare genetic disorder that forced them to eat cornstarch every few hours just got their first real treatment option. The FDA approved a one-time gene therapy that could free up to 2,500 Americans from a relentless routine that meant any missed dose risked seizures or death.
Two Philadelphia research teams just received millions in federal funding to develop one-dose gene therapies that could cure rare genetic diseases within five years. The breakthrough approach uses AI and new delivery methods to create treatments that work for multiple patients instead of requiring custom therapy for each person.
A paralyzed doctor who couldn't breathe on his own made a miraculous recovery from an autoimmune disease so rare he was only the 17th documented case worldwide. Now he's raising awareness and funds for the aqua therapy that saved him.
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.
Children born with a life-threatening immune disorder now have hope thanks to a newly approved gene therapy that could save their lives with a single treatment. The FDA just greenlit Kresladi, a breakthrough for severe LAD-1 that leaves kids defenseless against infections.
A groundbreaking initiative promises to make personalized genetic treatments affordable and accessible for millions of children with rare diseases. The "One to Millions" platform transforms how we develop and approve individualized therapies.
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