Watch along with us today for this historic meeting, the first-ever Advisory Committee meeting for a gene therapy specifically to treat #Duchenne.
Join today's live stream of the SRP-9001 Advisory Committee: parentprojectmd.org/may-12-fda-adv
Follow
Parent Project Muscular Dystrophy (PPMD)
@ParentProjectMD
Our mission is to #EndDuchenne, the most common fatal genetic disorder diagnosed in early childhood. #Duchenne
Parent Project Muscular Dystrophy (PPMD)’s Tweets
The FDA has shared live stream access information for the May 12th Advisory Committee for SRP-9001, Sarepta Therapeutics’s investigational #genetherapy for #Duchenne:
1
3
7
This Friday, May 12th, will be a historic day for the Duchenne community. It will be the day of the first-ever Advisory Committee meeting for a #genetherapy specifically to treat #Duchenne. Here's what you need to know before the meeting. parentprojectmd.org/what-you-need-
3
12
Race to end Duchene; with Team Parent Project Muscular Dystrophy, Five Boro Bike Ride for Charity
3
17
A big thank you 🙏🏻 to friends and colleagues for participating in #NYC 🗽 five borough Race to #EndDuchenne 🚴🚴♀ and for your generous donations to .
'Team Genocyclists, 22 strong' was captained by Abdullah "Bazi" Albanai 💪🏻
1
3
29
Ride for charity; five boro bike tour of NYC to raise funds for Duchenne muscular dystrophy with friends from Kuwait, Bahrain and UAE and #FiveBoro
1
3
12
In our written testimony to the SRP-9001 Advisory Committee, PPMD has submitted findings from our new #genetherapy preference study work with and : parentprojectmd.org/ppmd-submits-n
4
12
PPMD’s Knight Hacks is LIVE!
Have you been searching for an easier way to brush your teeth? A new way to organize your snack shelf that makes food easier to access? Accessible travel tips?
Explore some creative Hacks from members of the community: parentprojectmd.org/knight-hacks/
1
5
Sarepta has announced that the FDA Advisory Committee meeting for SRP-9001 will be held on May 12th. Join us in working together to ensure patient & scientific voices are represented. An informational webinar will take place Thursday, April 13 at 7 PM ET
4
5
15
Last night, PPMD & other leaders in the #Duchenne community met to plan how we can collaborate & work best together in preparation for the FDA Advisory Committee Meeting for SRP-9001. Learn how you can join us in working together:
1
2
Have you contacted your Members of Congress yet? The Senate deadline for the FY 2024 #Duchenne Funding Request closes this Friday. Please reach out to your members today to ask that they sign on before it closes! join.parentprojectmd.org/actionalert
1
While we await the date of the FDA advisory committee meeting for SRP-9001, PPMD will continue to work closely with our Duchenne community partners, as well as our partners at Sarepta, to prepare for this critical meeting. Learn how you can get involved:
2
4
Last week, 130+ PPMD Advocates from 28 different states participated in 131 meetings on Capitol Hill.
This week our Virtual PPMD Advocates will attend additional meetings. Take action today by contacting your members of Congress to amplify their message! join.parentprojectmd.org/actionalert
1
9
ACTION ALERT ‼️ #PPMDAdvocacy
Today, PPMD Advocates are BACK in person in Washington, DC to make their voices heard in the fight to #EndDuchenne! We need YOU to take action today to reinforce our message to Congress from home: join.parentprojectmd.org/actionalert
3
4
PPMD's Adult Advisory Committee (PAAC) had two awesome days of work, discussing their initiatives for the year ahead. Now, they are off to join the rest of our 130+ PPMD Advocates in preparing for meetings with Members of Congress as part of the 2023 #PPMDAdvocacy Conference!
4
19
Join PPMD and on Tuesday, March 7th at 12:00 PM ET for a community webinar discussing the current and future landscape of #Duchenne care and therapies.
Register now to receive a link to watch the webinar live or later on demand: neurocarelive.com/app/signup/030
1
Today is #RareDiseaseDay, a day to give voice to 300 million people living with rare diseases and their families, through education and advocacy. Will you commit to sharing your story and raising your voice? Join PPMD as a Virtual Advocate this March: parentprojectmd.org/advocacyconfer
1
12
Thrilled to announce the expansion of our Certified Duchenne Care Center Program with the pediatric certification of , our first certification in Arizona!
2
8
After 18 incredible years, Ryan Fischer will be transitioning off our team (he’s not going far, with plans to join PPMD’s Board of Directors in 2024!). We wish Ryan all the best. Please help us thank him for his years of contributions to our community!
3
2
13
Avidity Biosciences recently joined PPMD for a webinar to discuss the EXPLORE44™ clinical trial, an ongoing Phase 1/2 clinical trial evaluating AOC 1044 for the treatment of individuals with #Duchenne mutations amenable to exon 44 skipping. Recording:
1
2
It’s because of families like yours that we continue to move mountains on Capitol Hill. Register to join PPMD’s 2023 Advocacy Conference, March 5-7 in Washington, DC, and become an advocate for yourself and others living with Duchenne and Becker: parentprojectmd.org/advocacyconfer
1
5
An update on PPMD's newborn screening efforts after this afternoon's vote by the Advisory Committee on Heritable Disorders in Newborns and Children (ACHDNC): parentprojectmd.org/update-on-ppmd
3
Capricor recently joined PPMD for a community webinar to present recent data findings from the ongoing HOPE-2 open label extension trial and provide an update to the community on the ongoing HOPE-3 clinical trial. Recording now available:
4
PPMD is excited to announce a $500,000 programmatic investment in Myosana Therapeutics to support the continued development & translation of a non-viral gene therapy delivery platform aiming to slow skeletal muscle degeneration & heart failure in Duchenne.
1
REGENXBIO has announced that the Phase I/II AFFINITY DUCHENNE™ trial of RGX-202, an investigational gene therapy product for the potential treatment of #Duchenne, is now active and recruiting trial participants.
2
4
15
Excited to be back in person for PPMD’s 2023 Duchenne Healthcare Professionals Summit in NYC this week! 320+ clinicians, researchers & industry partners coming together from PPMD's 36 Certified #Duchenne Care Centers & other neuromuscular programs to ensure care is a priority.
4
10
PPMD has sent a request to the urging the agency to conduct a full review of ataluren (Translarna). Regardless of the outcome, the FDA’s review is critical for allowing U.S. families to make informed decisions about potential treatment options.
5
We are excited to launch PPMD Lighthouse Workshops - a safe space to build stronger connections & deepen insights about life and identity that goes beyond diagnosis & management. Women primary caregivers: Register to reserve your spot by Sunday, Jan 22!
1
THANK YOU! We are so grateful for your generosity & excited to announce that we have surpassed our goal to provide support to PPMD’s Cardiac Initiative and commitment to ACTION! Read more from PPMD's Pat Furlong about the impact your generosity will have: parentprojectmd.org/thank-you-for-
1
3
12
Today is the last day to double your gift and help us meet our goal of $400,000 to fund PPMD’s Cardiac Initiative and commitment to ACTION.
Help hearts grow stronger with every beat. Every dollar donated before midnight will be DOUBLED!
donate.parentprojectmd.org/give/442243/#!
2
3
got me headed down this path. She was ahead on the curve on the importance of cardiac evaluation and treatment. Give if you can. Pass it along if you can’t give. Happy holidays!
1
5
Double your gift today and help hearts like Taylor’s grow stronger with every beat: join.parentprojectmd.org/strongbeats
Thanks to the generosity of our community, every donation to support PPMD’s Cardiac Initiative and commitment to ACTION will be MATCHED through 12/31!
3
6
Linda Cripe, MD is a Pediatric Cardiologist at & one of the co-chairs of the Advanced Cardiac Therapies Improving Outcomes Network (ACTION) DMD subcommittee.
Watch the full video: youtu.be/Tgb9m_bgJgU
Support PPMD's Cardiac Initiative: donate.parentprojectmd.org/give/442243/#!
1
5
We are excited to announce that not only will we be back in person for PPMD’s 2023 Advocacy Conference, March 5-7th in Washington, DC, but this year will mark our first HYBRID Advocacy Conference with both in-person and virtual advocate options. Join us:
1
6
WATCH: Pat Chats with Chet Villa, MD about the Advanced Cardiac Therapies Improving Outcomes Network (ACTION) and #Duchenne cardiac care and research. Your support will help us make hearts Stronger With Every Beat: donate.parentprojectmd.org/give/442243/#!
1
4
Protect Your Lungs This Winter: Don't miss our webinar today, Dec. 8 at 1 PM ET as we hear from three expert pulmonologists as they discuss respiratory management in #Duchenne to help ensure you are as prepared as possible this winter season. join.parentprojectmd.org/webinar2
1
2
Today is the 30th ! PPMD is so excited to have been nominated and chosen as one of this year’s #ICAPCharityDay beneficiaries. Funds donated as a result of Charity Day will support PPMD’s mission to #EndDuchenne. icapcharityday.com/charities/2022
1
1
Protect Your Lungs This Winter: Join PPMD this Thurs, Dec. 8 at 1 PM ET for a webinar as we hear from three expert pulmonologists as they discuss respiratory management in #Duchenne to help ensure you are as prepared as possible this winter season. join.parentprojectmd.org/webinar2
1
3
"The ability to view in the Interchange population-level data from our clinic compared to other #Duchenne & Becker clinics in the CDCC Network is incredibly valuable to advancing the science.”
-Dr. Veerapandiyan
More about PPMD's EHR Study:
prnewswire.com/news-releases/
2
3
On the latest episode of PPMD's Living Duchenne podcast, we talk with the Wolf family & Coach Keith Otterbein of to hear how the unlikely connection between #Duchenne and college football sparked a lifelong friendship.
Now available wherever you get your podcasts!
1
1