PPMD's Blog – December 2017 Archive (10)

Two days left to reach our goal



I am a 22 year old living with Duchenne, and opportunities for clinical trial participation are few and far between. It is one of the frustrating realities of my diagnosis and my age. Although it has never swayed my commitment to this community, I have to be honest. It can be discouraging at…

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Added by PPMD on December 30, 2017 at 10:03am — No Comments

Great News: Match extended by PPMD board!

I have some exciting news: Your response to Sean and Mindy Rice’s generous matching gift of $200,000 has been incredibly inspiring to the Parent Project Muscular Dystrophy board. You’ve shown us that PPMD’s Gene Therapy Initiative matters to you, and we want to keep up the momentum.…

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Added by PPMD on December 26, 2017 at 3:00pm — No Comments

Gene therapy gives me the courage to hope

My son Bazi is four years old and has Duchenne muscular dystrophy. It's been over a year now, and this sentence is still extraordinarily hard for me to write.



When we got the diagnosis, my emotions shifted between paralyzing fear, incredible…

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Added by PPMD on December 22, 2017 at 11:30am — No Comments

We're doubling your donation for gene therapy

 

When our son, Jonathan, was diagnosed with Duchenne, we didn’t know where our family would find the strength to fight. But the PPMD community has always shown us that we are never alone in our fight to end Duchenne.



Because…

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Added by PPMD on December 19, 2017 at 2:56pm — No Comments

Understanding Gene Therapy: The Screening Process

With advancements made in Duchenne gene therapies now coming to fruition, a number of questions are being asked about who may qualify for the upcoming trials.

 

While the inclusion criteria of each trial will be different based on age, mutation (in some cases), and functional abilities, one of the criteria for participating in…

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Added by PPMD on December 13, 2017 at 4:00pm — 1 Comment

PTC Therapeutics Shares Update on Ataluren Formal Dispute Resolution Request Process

PTC Therapeutics provided a brief update on the Formal Dispute Resolution Request process for ataluren.

Read the update from PTC:

Dear Duchenne Community,



As we enter the holiday season, I wanted to thank the community for all your support throughout the regulatory…

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Added by PPMD on December 13, 2017 at 11:00am — No Comments

Santhera Update for Duchenne Community on SIDEROS Study

Santhera has provided an update to the Duchenne community, which contains updates on the SIDEROS study, educational events, respiratory guidelines/care, and more. Click here to view the update.…

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Added by PPMD on December 11, 2017 at 4:30pm — No Comments

PPMD Signs Letter to Save CDC

PPMD joined 153 members of the CDC Coalition and other supporting state and national organizations in urging Congressional leaders to provide at least $7.8 billion for the Centers for Disease Control and Prevention’s programs as part of the final FY 2018 Labor, Health and Human Services, Education and Related Agencies Appropriations bill and other final FY…

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Added by PPMD on December 7, 2017 at 12:20pm — No Comments

Duchenne/Becker and FSH Muscular Dystrophies Receive ICD-10 Codes

Parent Project Muscular Dystrophy, FSH Society Lead Effort to Obtain

Critical Diagnostic Classification Standard

 

Parent Project Muscular Dystrophy (PPMD)  along with collaborators the…

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Added by PPMD on December 7, 2017 at 8:30am — No Comments

PPMD Designates UCSF Benioff Children's Hospital San Francisco a Certified Duchenne Care Center

Today Parent Project Muscular Dystrophy (PPMD) named UCSF Benioff Children’s Hospital San Francisco (UCSF) our 18th Certified Duchenne Care Center.

 

PPMD’s Certified Duchenne Care Center Program, which…

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Added by PPMD on December 6, 2017 at 9:30am — No Comments

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