FDA OKs First ‘Targeted Treatment’ for Rare DMD Mutation
The US Food and Drug Administration (FDA) has approved the antisense oligonucleotide casimersen (Amondys 45,...
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Tag: Personalized Medicine
Breaking the ‘stalemate’ in the most common soft tissue sarcoma in children: First randomized clinical trial to show positive results in rhabdomyosarcoma since 1974
A phase 2 clinical trial has found that combining a molecular targeted drug called temsirolimus...
Infant with deadly leukemia saved by drug for adult liver cancer: Identifying genetic mutations may unlock cure for challenging malignancies
UCSF Benioff Children's Hospitals have successfully treated a months-old infant with a rare childhood leukemia...
Genetic basis for re-experiencing symptoms in PTSD: Analysis included more than 165,000 military veterans
A study based on the Million Veteran Program of the U.S. Department of Veterans Affairs...
Beyond finding a gene: Same repeated stretch of DNA in three neurodegenerative diseases
Families living with extremely rare neurodegenerative diseases finally received an answer to the cause of...
Linking phenotypes to genotypes: A newly devised gene-editing strategy
The power and convenience of modern-day word-processing programs, like Microsoft Word, have revolutionized our daily...
Gene networks reveal transition from healthy to failing heart
Scientists investigating heart failure have been limited to studying diseased heart tissue in the lab...
Are we using biologic therapy properly?
The introduction of infliximab (Remicade), the first biologic therapy approved for the treatment of inflammatory...
Genetic inequity towards endocrine disruptors
Phthalates, one of the most common endocrine disruptors, are commonly used by industry in many...
Big data reveals hidden subtypes of sepsis
Much like cancer, sepsis isn't simply one condition but rather many conditions that could benefit...