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St. Jude Children’s Research Hospital: A beacon of hope
KTVI via Yahoo News· 5 hours ago“If you have this disease or genetic disorder…if your child is sick in St. Louis or sick in Memphis,...
Congenital muscular dystrophy: Symptoms, causes, and more
Medical News Today· 4 days agoCongenital muscular dystrophy (CMD) is a group of muscle disorders that can appear soon after birth....
AI blood test could detect Parkinson's disease up to 7 years before symptoms: 'Particularly...
Fox News via Yahoo News· 13 hours agoA new blood test could reveal Parkinson’s diagnoses up to seven years before symptoms emerge,...
Taysha links gene therapy to motor gains in small Rett study
FierceBiotech· 4 days agoTaysha Gene Therapies has unfurled more data in the developmental disorder Rett syndrome, linking...
Gene editing technology is revolutionizing disease treatment | The Excerpt
USA TODAY via Yahoo News· 1 day agoSo we know that everyone has millions of different genetic variants. This is what makes us different or unique as people. And so when...bring these therapies to patients for ...
12 recent scientific breakthroughs
The Week via Yahoo News· 4 days agoA study published in the journal Nature found that a drug called antisense oligonucleotide allowed human neurons to develop normally despite carrying a ...
FDA Expands Approval of Gene Therapy for Patients with Duchenne Muscular Dystrophy
The Woonsocket Call· 1 day agoToday, the U.S. Food and Drug Administration expanded the approval of Elevidys (delandistrogene moxeparvovec-rokl), a gene therapy for the treatment of Duchenne muscular dystrophy (DMD) for ...
Family's genes offer clue to delaying onset of Alzheimer's
Napa Valley Register· 21 hours agoScientists studying a family plagued by early-in-life Alzheimer’s found some carry a genetic oddity...
Drug approved as sickle cell disease treatment may help stabilize vision in rare genetic disease
Medical Xpress· 5 days agoIn a new study, the team reports that a drug approved by the FDA for another condition may stabilize...
New blood test could predict Parkinson’s disease 7 years before symptoms
MedicalNewsToday via AOL· 4 days agoAlessandro...Disease, prodromal symptoms include: Constipation Loss of smell Possible REM-sleep...