Matt Walsh

Community Engagement Coordinator for Parent Project Muscular Dystrophy. Leading the fight to #EndDuchenne.

[AnnemiekeAartsma-Rus @oligogirl]

Tina Duong presenting upper limb function and patient reported outcome measures that were developped WITH patients #PPMDConnect

[Alan Chaulet ♿✈ @AlanChaulet]

Excited to watch the @ParentProjectMD #PPMDconnect conference. Wish I was there this year! https://t.co/0WtSCNJV0F

[AnnemiekeAartsma-Rus @oligogirl]

Today at #ppmdconnect whole day with companies who do or plan DMD trials.Remember when we got excited about tests in mice rather than cells?

[The Duchenne Registry @DuchenneReg]

Pipeline with Raxone #ppmdconnect

[The Duchenne Registry @DuchenneReg]

https://t.co/1ftqDelg1H provides more detail #ppmdconnect

[The Duchenne Registry @DuchenneReg]

Domagrozumab will have open label once period 1 &2 have been completed by participants #ppmdconnect

[Matt Walsh @MattPPMD]

PF-06252616 FAQ Page 27 #PPMDconnect

[The Duchenne Registry @DuchenneReg]

You can also check our FAQ's on our website and in your handouts https://t.co/IdCbDtfHrQ

[Matt Walsh @MattPPMD]

Dr. Michael Binks from @pfizer now talking about Domagrozumab at the #PPMDconnect conference

[Matt Walsh @MattPPMD]

Visit https://t.co/PsmhaK0AYN to learn about clinical trials and the @DuchenneConnect table to talk to our Genetic Counslors #ppmdconnect

[The Duchenne Registry @DuchenneReg]

Ongoing study in Duchenne BMS089 #ppmdconnect

[Matt Walsh @MattPPMD]

Criteria BMS 089 trial: inclusion criteria: ages 5-11, corticosteroid use past 6 months, 4 stair climb less <= 8 seconds #PPMDconnect

[Matt Walsh @MattPPMD]

Preliminary Phase1 of BMS 089: shows increased thigh muscle volume in heathy volunteers #PPMDconnect

[The Duchenne Registry @DuchenneReg]

BMS 908 phase I study generally safe and well tolerated, most common effects mild rash and site rxn #ppmdconnect

[The Duchenne Registry @DuchenneReg]

Myostatin #ppmdconnect

[Matt Walsh @MattPPMD]

Anti-myostatin antibody improves the phenotype of mdx mouse -increased motor activity, decreased serum CK #PPMDconnect

[Matt Walsh @MattPPMD]

Removal of myostatin (GDF-8) in mice results in increased skeletal muscle, bone density #PPMDconnect

[Matt Walsh @MattPPMD]

Next to present is Leslie Jacobsen, MD with BMS at #PPMDconnect

[The Duchenne Registry @DuchenneReg]

Sarepta conclusion slide #ppmdconnect

[The Duchenne Registry @DuchenneReg]

#DRSC hope is to have shorter smaller trials, possibly reduced placebo arm size, greater agreement researchers, regulators, pts #PPMDConnect