Grace Pavlath

Vice President Translational Research Solid Biosciences , #Gene Therapy, #Neuromuscular, #RareDisease

[Barry Byrne @barrybyrne1]

Bo Cumbo jumps from the top commercial post at Sarepta to the helm of a gene therapy startup with some influential backers, big plans and $107M – Endpoints News https://t.co/pdOPN7L1jm

[Eldad Tzahor @Tzahore]

Sad day for the muscle community with the passing of Prof. David Yaffe from our department @WeizmannScience David (born in 1929) has pioneered culture methods for muscle stem cells including the famous C2 cells.

[Brian Tyler Cohen @briantylercohen]

The Trump campaign seriously just issued a cease and desist for this ad so I'm doing my part and sharing it as widely as humanly possible.

[Alison Lynch-Miller @AlisonLynchMil1]

#LetTheScientistsLead If this was a war and the doctors took over the planning, would that make any damn sense? Medical professionals are merely fodder in Trump’s mismanagement. Let’s stand up and say enough, LetTheScientistsLead!

[Katie Baca-Motes @BacaMotes]

“Doctor groups are recommending testing and isolation for people who lose their ability to smell and taste, even if they have no other symptoms.” https://t.co/x3W9186weE

[Grace Pavlath @GPavlathScience]

Fascinating implications of chimerism after bone marrow transplant for forensics https://t.co/77cMcln5vK

[Grace Pavlath @GPavlathScience]

@barrybyrne1 Agre! Favorite movie of the summer.

[Lynn O’Connor Vos @lynnvos]

Very interesting read given #genetherapy #geneediting conversations had at yesterday's #MDAconference https://t.co/1QAW7OT5fS #CRISPR #Cancer

[Grace Pavlath @GPavlathScience]

Speech may become an important outcome measure in future clinical trials in ALS. Great talk by Jeremy Shefner at #MDAConference #als

[Lynn O’Connor Vos @lynnvos]

MDA National Ambassadors Justin, Tana and Faith tried out the new @Microsoft adaptive xbox controller - a 1st xbox experience for Faith. Justin, a strategic and steady driver in the game, mentioned excitement for the ability to customize the controller so anyone can make it work.

[Lynn O’Connor Vos @lynnvos]

Dr. Janet Woodcock at the #MDAconference @US_FDA

[Dr. Jodi Wolff @drjodiwolff]

Fantastic morning ⁦@MDAorg⁩ learning about congenital muscle diseases with Jim Dowling, Reghan Foley & Carsten Bonneman #MDAconference #coll6 #mtm1 #myotubularmyopathy #cmd

[Grace Pavlath @GPavlathScience]

Lindsay Alfano of @nationwidekids reminds us that selection of outcome measures is not “one-size-fits-all” - we must continue to re-evaluate the outcome as treatments emerge & ‘natural history’ is redefined. Great Outcome Measures talk at #MDAConference

[Grace Pavlath @GPavlathScience]

In yesterday's session on Gene–Targeting Therapies, Dr Robert Brown at the UMass Med School shared progress of a pilot human clinical trial using a gene-targeting therapy for a common mutation in #ALS. #MDAconference

[Lynn O’Connor Vos @lynnvos]

We at MDA are truly honored to receive the @ASGCTherapy Sonia Skarlatos Public Service Award for significant contribution to gene and cell therapy. Congrats to our entire Scientific team here @MDAorg @GPavlathScience @AHaidetPhillips @LOrlandoScience @Hagerty_Laura #GeneTherapy

[Laura Hagerty @Hagerty_Laura]

Kicking off the 2019 MDA Clinical and Scientific Conference with a meeting of the MDA Care Center Director physicians. Great discussion underway with the docs trading learnings from across their centers. @MDAorg

[Grace Pavlath @GPavlathScience]

Fantastic kick off to the #MDAConference! Today's MDA Scholars session invited 4 early stage scientists to present their research on neuromuscular diseases -- research made possible with Development Grants from @MDAorg.

[Grace Pavlath @GPavlathScience]

So excited to see an innovative collaboration between @AmicusRx and @PennMedicin,e which will explore novel gene therapy approaches for treating rare metabolic diseases including #Pompe, #Fabry, and #CDKL5! #RareCompany [https://t.co/LjptKooPEl]

[Grace Pavlath @GPavlathScience]

Latest round of research funding from MDA: https://t.co/osxJhZveai