Abby Bronson's Blog (27)

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…


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 clinical trial design.…


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 Opinion…


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

PPMD is Initial Partner in OnPAR — Online Partnership to Accelerate Research

The National Institutes of Health (NIH) is one of the biggest funders of medical research in the US, and projects it has funded have touched almost every Duchenne therapeutic in development now in some way. In FY2016, the NIH is investing nearly $32.3 billion in medical research, and of…


Added by Abby Bronson on March 24, 2016 at 9:30am — No Comments

Gene Therapy Today: Notes from PPMD’s Duchenne Gene Therapy Forum

Gene therapy.

Those two words have been tossed around for some time, starting in the late 1990s when several gene therapy trials started, but ended with fatal results. Now, when the field has learned a lot from those initial results and with optimized technologies, we come back to gene therapy as a viable option for genetic diseases. This renewed…


Added by Abby Bronson on March 8, 2016 at 12:00pm — 4 Comments

Finding Routes to Duchenne Drug Approvals

It seems that everyone is looking for the newest molecule, the newest therapy, the newest technique that will halt the progression of Duchenne. This is good and we should continue on this journey. But none of these novel treatments will get to patients without first going through a rigorous evaluation in clinical trials.



Added by Abby Bronson on February 20, 2016 at 10:28am — No Comments

PPMD Announces Gene Therapy Forum

This year’s already action-packed Advocacy Conference, February 21-23 welcomes a special Gene Therapy Forum on Monday, February 22 from 9am to 1pm at The Mayflower Hotel in D.C. Hosted by…


Added by Abby Bronson on February 10, 2016 at 10:30am — 2 Comments

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