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Abby Bronson's Blog (24)

Gene Transfer in the Spotlight

On September 28 and 29, I had the privilege of attending an in-person Wellstone Center meeting that Dr. Eric Olson and his Wellstone team at UT Southwestern Medical Center hosted in Dallas, TX. The breadth and depth of what the Wellstone Centers have worked on over the years…

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Added by Abby Bronson on October 11, 2017 at 12:30pm — 2 Comments

Webinar Q&A: Gene Therapy Part 2 – Nationwide Children's Hospital

This fall, PPMD is presenting a gene therapy webinar series with companies and institutions who are developing therapies for Duchenne that are commonly referred to as gene therapy, including…

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Added by Abby Bronson on October 2, 2017 at 1:30pm — No Comments

PPMD Research Update: September 2017

As you know by now, September is Duchenne Action Month. And…

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Added by Abby Bronson on September 21, 2017 at 12:00pm — No Comments

FDA Announces Approval of First Gene Therapy in the United States

Yesterday, Kymriah, a gene therapy made by Novartis that treats an aggressive type of leukemia was approved for children and young adults. The new commissioner of the FDA, Dr. Scott Gottlieb,…

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Added by Abby Bronson on August 31, 2017 at 1:00pm — No Comments

Webinar Q&A: Gene Therapy for Duchenne

With trials in gene therapy on the horizon, and terms like "cassette", "construct", "promoter", and "transduce" being thrown around, we want to ensure that you, our Duchenne Community, has all the knowledge you need to navigate the world of gene therapy.



Earlier this…

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Added by Abby Bronson on August 23, 2017 at 8:00pm — No Comments

PPMD Research Update: June 2017

An Overview of Your Impact in Action



We are less than a week away from PPMD’s Annual Connect Conference, where researchers and industry from around the world will provide…

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Added by Abby Bronson on June 23, 2017 at 2:00pm — No Comments

2017 DRSC Annual Meeting

On May 9, the Duchenne Regulatory Science Consortium (DRSC) held its Annual Meeting in Crystal City, VA. In the audience of about 40 people, there were industry and academic Consortium members, potential industry members, five FDA representatives, two NIH representatives, C-PATH representatives, and Buddy Cassidy, the DRSC…

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Added by Abby Bronson on May 16, 2017 at 3:00pm — No Comments

PPMD's Duchenne Drug Development Roundtable Meeting Series, Part 2: Optimizing Clinical Trials

This week, PPMD convened Duchenne industry partners, experts in adaptive trial design and biostatistics, and innovative partners from outside of the Duchenne world who’ve revolutionized their clinical trial spaces for the second meeting of our Duchenne Drug Development Roundtable's 3-part meeting series, focusing on Clinical…

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Added by Abby Bronson on March 3, 2017 at 10:00am — No Comments

Duchenne Research Update: February 2017

 

An Overview of Your Impact in Action



PPMD Grant Awards in the last Quarter of 2016 and first part of 2017.

 

PPMD Launches Long-Term…

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Added by Abby Bronson on February 24, 2017 at 1:29pm — No Comments

PPMD’s Duchenne Drug Development Roundtable: Bringing Industry Together to Accelerate Research

Better. Faster. Now.

 

How do we apply the ‘regulatory learnings’ of the past 18 months back into our robust Duchenne pipeline to ensure as many effective therapies yield from our pipeline as possible?

Under the leadership of PPMD's Duchenne Drug Development Roundtable (DDDR), our community is determined to figure that out. Today is the first of a…

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Added by Abby Bronson on February 1, 2017 at 10:00am — No Comments

DOUBLE your investment in the future

Help us raise $250,000 by 12/31!…



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Added by Abby Bronson on December 20, 2016 at 12:00pm — No Comments

PPMD Is Seizing This Moment to Explore the Potential of CRISPR/Cas9 in Duchenne

It is the holidays – time for giving and laughter and hope. Hope comes in many forms, but I would imagine that for many reading this blog, hope comes in the form of a cure for Duchenne.  

Could CRISPR/Cas9 offer hope for treating Duchenne? 

As background, CRISPR/Cas9 is a…

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Added by Abby Bronson on December 8, 2016 at 3:30pm — No Comments

Duchenne Research Update: October 2016

When I first joined the Duchenne community in 2009 at Children’s National Medical Center, I remember thinking that I was walking into a critical juncture in Duchenne history. There were three compounds in the clinic (drisapersen, ataluren, and eteplirsen aka Exondys…

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Added by Abby Bronson on October 18, 2016 at 1:00pm — No Comments

PPMD Awards $200,000 Grant to Joshua Selsby, PhD of Iowa State University

Quercetin /ˈkwɜːrstn/ is a flavonol found in many fruits, vegetables, leaves and grains. It can be used as an ingredient in supplements, beverages, or foods. Source:…

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Added by Abby Bronson on September 8, 2016 at 9:43am — No Comments

Understanding Gene Therapy & CRISPR/Cas9: Upcoming Clinical DMD Symposium

Last month, PPMD welcomed Dr. Eric Olson from UT Southwestern Medical Center to discuss the basics of gene editing and the potential permanent correction of Duchenne mutations with CRISPR/Cas9. If you missed it live, check out the…

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Added by Abby Bronson on August 12, 2016 at 10:09am — No Comments

Understanding Gene Therapy & CRISPR/Cas9

You’ve seen the headlines and heard the name, now find out more about the latest in gene therapy, CRISPR/Cas9, and what these technologies could mean for Duchenne during three different opportunities that promise to provide an in-depth analysis:



  • Gene Therapy and…
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Added by Abby Bronson on June 16, 2016 at 11:04am — No Comments

PPMD Awards University of Washington Grant to Support Technology to Help Optimize Gene Therapy

The promise of gene therapy continues to build momentum, in Duchenne and also in other conditions. Much has been learned about the immunological and delivery challenges of the approach since gene therapy’s early attempts in the 1990s.

In Duchenne, some of the early work in gene therapy ended with immune system reactions to the new dystrophin that was expressed by the transfected gene. But this was not…

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Added by Abby Bronson on June 9, 2016 at 12:00pm — No Comments

DRSC Definitions

A couple of months ago we posted a write up about some of the efforts PPMD is undertaking to help improve…

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Added by Abby Bronson on May 16, 2016 at 8:40am — 2 Comments

Update on Gene Replacement/Repair Strategies for Duchenne Muscular Dystrophy

There has been a flurry of press recently on gene therapy in Duchenne muscular dystrophy and CRISPR technology. With this barrage of information, PPMD wanted to take a deeper dive into gene therapy as a potential treatment for Duchenne.

 

An Expert…

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Added by Abby Bronson on March 25, 2016 at 9:30am — No Comments

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