Carrie Miceli

[Carrie Miceli @MiceliCarrie]

@TerriEllsworth Fingers crossed

[Carrie Miceli @MiceliCarrie]

@TheEllenShow How

[Carrie Miceli @MiceliCarrie]

@FDArecalls

[CDMD at UCLA @cdmducla]

Mint pmi restored dystrophin functional because other dgc components restored. Agree #MakeDuchenneHistory #DNADay16

[Parent Project Muscular Dystrophy (PPMD) @ParentProjectMD]

We don't know the level of dystrophin needed to deliver a benefit - Dr. Woodcock #MakeDuchenneHistory

[Parent Project Muscular Dystrophy (PPMD) @ParentProjectMD]

Dr. Woodcock giving background of how FDA has used flexibility when approving medicines #MakeDuchenneHistory

[CDMD at UCLA @cdmducla]

@US_FDA flexibility is best demonstrated through careful review of totality of data. More uncertainty is tolerated #MakeDuchenneHistory

[Parent Project Muscular Dystrophy (PPMD) @ParentProjectMD]

From beginning, historical control have always been included #MakeDuchenneHistory

[CDMD at UCLA @cdmducla]

Historical controls are and can be used for regulatory approval Dr temple

[CDMD at UCLA @cdmducla]

It's appropriate to be critical of external control groups. Multiple ex is better (and supports a positive drug effect) #MakeDuchenneHistory

[Parent Project Muscular Dystrophy (PPMD) @ParentProjectMD]

Great looking guys! #MakeDuchenneHistory #endDuchenne https://t.co/APWBivBpOp

[Parent Project Muscular Dystrophy (PPMD) @ParentProjectMD]

Dr. Temple reviewing the issues in other studies with historical controls #MakeDuchenneHistory

[Jett Foundation @JettFoundation]

Even the overflow room is overflowing to listen to Edward Kaye, MD, CMO, (Interim CEO) $SRPT #believeyoureyes

[Barack Obama @BarackObama]

"Improving gender equality would add at least $2.1 trillion to U.S. gross domestic product by 2025." https://t.co/YHZW8QPEpR #EqualPayDay

[Barack Obama @BarackObama]

It's time. #EqualPayDay

[CDMD at UCLA @cdmducla]

You are cordially invited to Dealing For Duchenne! - R.S.V.P. by April 30th for May 14th #D4D https://t.co/mIS6wBLGoO

[Barack Obama @BarackObama]

Retweet if you agree. #EqualPayDay

[April Pyle @apyle]

Imprecision Medicine: A One-Size-Fits-Many Approach for Muscle Dystrophy: Cell Stem Cell https://t.co/wJAHvFi0cq

[d//y//l//a//n @FistFightingDMD]

"Intelligence is the ability to adopt to change" -Stephen Hawking

[Terri Ellsworth @TerriEllsworth]

True story-almost 5 yrs later Billy is still walking well at 15 1/4 yrs old because of #Eteplirsen $SRPT #DMD #FDA https://t.co/Iemy7w2Rer