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Searching for Surrogates: The Effort to Speed Up Drug Trials

Identifying surrogate endpoints for use in clinical trials that strongly predict clinical outcomes such as improved survival and quality of life is challenging.

United statesNational institutes of healthSan franciscoAmaru kishanHiddol heerspinkVincent plancheJuliaa beaverCarol taylor burdsLos angelesKarin hoelzerFabio confortiRichard pazdurAnushka waliaEmersony chenGinny beakesFady ghali

Searching for Surrogates: The Effort to Speed Up Drug Trials

Identifying surrogate endpoints for use in clinical trials that strongly predict clinical outcomes such as improved survival and quality of life is challenging.

United statesNational institutes of healthSan franciscoFabio confortiVincent plancheFady ghaliKarin hoelzerGinny beakesCarol taylor burdsAmaru kishanRichard pazdurHiddol heerspinkAnushka waliaLos angelesEmersony chenJuliaa beaver

Rady s newborn screening program prepares to expand

Rady Children's plans to expand genetic newborn screening efforts that test for 419 treatable conditions rather than the 80 currently required in California.

United statesSan diegoStephen kingsmoreLe bonheurKarin hoelzerUniversity of tennesseeScripps healthColumbia universityRady children institute for genomic medicineCornell universityNational organization for rare disordersRady childrenGenomic medicineBoston childrenNational organizationRare disorders

[Webinar] The NCATS Alliance: Speeding the Process of Bringing New Treatments to Patients - March 15th, 1:00 pm - 2:00 pm ET | Manatt, Phelps & Phillips, LLP

Featuring Francis Collins, M.D., Ph.D., Senior Advisor to President Biden and Former Director of the National Institutes of Health, and Joni Rutter, Ph.D., NCATS Director, Plus a Panel.

United statesFrancis collinsJonny mcphersonJoni rutterPaul melmeyerRon bartekDiana pankevichKarin hoelzerRachel sherJonil rutterKristin stephensonKristin schneemanChristopher austinMuscular dystrophy associationGovernment affairs publicNational center

Lawmakers Must Be Educated About AADC, Other Rare Disease: NORD

As the 118th U.S. Congress opens, the National Organization for Rare Disorders argues it is important to engage lawmakers about health policy.

Karin hoelzerUs congressRare action network or advocacy taskforceNational organization for rare disordersMedicare medicaid servicesRare disease advisory councilDrug administrationNational organizationRare disordersCapitol hillAction networkAdvocacy taskforceMedicaid servicesRare disease advisory

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