Recapping the FDA-NIH Dystrophin Methodology Workshop
PPMD CEO, Dr. John Porter, recaps yesterday's FDA and NIH dystrophin methodology workshop. This was an important moment for the Duchenne community and PPMD was honored to be a part of it. Thank you to all the families who came to the meeting. (Source: Parent Project Muscular Dystrophy)
Source: Parent Project Muscular Dystrophy - March 21, 2015 Category: Neurology Source Type: news
Dollars Destroying Duchenne
PPMD loves creative, out-of-the-box fundraisers that raise awareness. This new, annual event from Davenport, FL is a great example of something you can do in your community. Read organizer Diana Rodriguez’s story and don’t hesitate to reach out to us with your own ideas. PPMD has all the tools you need to create a truly memorable and important event! (Source: Parent Project Muscular Dystrophy)
Source: Parent Project Muscular Dystrophy - March 18, 2015 Category: Neurology Source Type: news
PPMD Participating in FDA-NIH Dystrophin Methodology Meeting on Friday, March 20
PPMD is grateful that the FDA and NIH are willing to discuss dystrophin and have opened their doors to include our community leaders.
If you would like to attend Friday’s public scientific workshop, see details here: http://1.usa.gov/1GYNSpV. If you cannot attend but would like to watch a live stream of the meeting, view this link Friday morning to attend virtually: http://1.usa.gov/1GYNSpV. (Source: Parent Project Muscular Dystrophy)
Source: Parent Project Muscular Dystrophy - March 18, 2015 Category: Neurology Source Type: news
Registration for PPMD's 2015 Connect Conference is Now Open
Parent Project Muscular Dystrophy’s Annual Connect Conference is a unique convergence of industry partners, scientific leaders, medical providers, people living with Duchenne, and their families. This exceptional meeting has grown to be recognized worldwide as the foremost Duchenne muscular dystrophy meeting. But more than that, it is a way for families affected by Duchenne to connect with each other. To build support networks. To realize no one is on their own in the fight to end Duchenne.
This year, 2015, marks our 21st Connect Conference. With the Duchenne landscape changing in so many ways, this year’s meeting wil...
Source: Parent Project Muscular Dystrophy - March 18, 2015 Category: Neurology Source Type: news
Spring Forward: Bringing the MD-CARE Act to Life
Despite great odds in a political climate that was stacked against us – we achieved what was considered to have been impossible. After months of outreach to congressional champions and visits to Capitol Hill, the MD-CARE Act Amendments were passed and signed into law in the end of 2014.
Now what? Well… now the real work of implementation actually begins. On Tuesday, March 17, the Muscular Dystrophy Coordinating Committee (MDCC) will be taking the first formal step to bring the MD-CARE Act Amendments to life – and our community will officially Spring forward! (Source: Parent Project Muscular Dystrophy)
Source: Parent Project Muscular Dystrophy - March 18, 2015 Category: Neurology Source Type: news
What is Epicatechin?
Epicatechin. It’s a word that is starting to pop up more and more in the Duchenne community. But was is it? PPMD helps explain this exciting potential therapy now in the early stages of development. (Source: Parent Project Muscular Dystrophy)
Source: Parent Project Muscular Dystrophy - March 18, 2015 Category: Neurology Source Type: news
Urge Congress to Sign Duchenne Funding Letter
PPMD’s Annual Advocacy Conference in February was a huge success! Now the important follow up needs to happen. Please take one minute to send an important action alert to urge Congress to continue focusing federal resources on Duchenne. The deadline for House members to sign onto the funding letter is March 20, 2015, so please take action today! (Source: Parent Project Muscular Dystrophy)
Source: Parent Project Muscular Dystrophy - March 16, 2015 Category: Neurology Source Type: news
Recall Notice for Deflazacort 30mg
If you receive this recall notice and/or think that you may have an affected batch (J021 [expiry 30/06/2017] or J028 [expiry 30/09/2017]), you should contact your provider and ask them to replace it with a prescription for prednisone to take until you get your new batches of deflazacort. Do not take deflazacort that is part of the affected batch. As more information becomes available, we will share it with the community immediately. If you have any further questions call your neuromuscular provider. (Source: Parent Project Muscular Dystrophy)
Source: Parent Project Muscular Dystrophy - March 16, 2015 Category: Neurology Source Type: news
PPMD Names Stanford Comprehensive Neuromuscular Clinic at Lucile Packard Children ’s Hospital a Certified Duchenne Care Center
Dr. Day has been a longtime partner in the Duchenne community, as an advocate for both optimal care in the patient community and as a leading researcher in muscular dystrophy. The clinical team he has assembled at Stanford is a shining example of the skill, compassion, and understanding we look for in certifying clinics. < div class="feedflare" > < a href="http://feeds.parentprojectmd.org/~ff/ppmd?a=22gjO_jhoyU:AHGO8cvXdkA:yIl2AUoC8zA" > < img src="http://feeds.feedburner.com/~ff/ppmd?d=yIl2AUoC8zA" border="0" > < /img > < /a > < a href="http://feeds.parentprojectmd.org/~ff/ppmd?a=22gjO_jhoyU:AHGO8cvXdkA:qj6IDK7rITs" > < i...
Source: Parent Project Muscular Dystrophy - March 16, 2015 Category: Neurology Source Type: news
PPMD Names Stanford Comprehensive Neuromuscular Clinic at Lucile Packard Children’s Hospital a Certified Duchenne Care Center
Dr. Day has been a longtime partner in the Duchenne community, as an advocate for both optimal care in the patient community and as a leading researcher in muscular dystrophy. The clinical team he has assembled at Stanford is a shining example of the skill, compassion, and understanding we look for in certifying clinics. (Source: Parent Project Muscular Dystrophy)
Source: Parent Project Muscular Dystrophy - March 16, 2015 Category: Neurology Source Type: news
Dystrophin 101 Webinar Recording
The recording of our "Dystrophin 101" webinar is now available. Watch now to learn more about the basic biology of dystrophin, how dystrophin is measured in both preclinical studies and clinical trials, and the need for and role that dystrophin analysis play in clinical trials of novel agents designed to exert their therapeutic effects through an increase in dystrophin levels. Our speakers also discussed what is required in order to have dystrophin considered as a surrogate. (Source: Parent Project Muscular Dystrophy)
Source: Parent Project Muscular Dystrophy - March 16, 2015 Category: Neurology Source Type: news
We Need YOU to Help Us With the Development of the Solid Suit
PPMD is partnering with some really smart folks to develop wearable technology to enhance strength for people with Duchenne and Becker muscular dystrophy. The Solid Suit is envisioned to be a soft, wearable, assistive device. Conceptually, it could be worn under one’s clothing – next to his/her skin (picture Spider-Man’s suit). The Solid Suit team has been hard at work over the last few months, and we are now turning to the experts with our community – YOU – to help inform the design of the project through a community survey. (Source: Parent Project Muscular Dystrophy)
Source: Parent Project Muscular Dystrophy - March 2, 2015 Category: Neurology Source Type: news
Important New Duchenne Steroid Protocol: Download the " PJ Nicholoff Steroid Protocol "
Families living with Duchenne muscular dystrophy are aware that maintaining regular doses of corticosteroids is important, but “why” and “how” can be confusing. We have worked together with experts to develop a tool that hopes to simplify these issues. Keep this protocol available to you, and your medical providers, in case of a medical emergency or admission.
Important steroid protocol topics covered:
- Corticosteroid conversions/equivalent doses
- Appropriate corticosteroid stress doses
- Recommendations for corticosteroid withdrawal
- Symptoms of adrenal crisis
- Tests that can help diagnose adrenal crisis
- Def...
Source: Parent Project Muscular Dystrophy - March 2, 2015 Category: Neurology Source Type: news
Important New Duchenne Steroid Protocol: Download the "PJ Nicholoff Steroid Protocol"
Families living with Duchenne muscular dystrophy are aware that maintaining regular doses of corticosteroids is important, but “why” and “how” can be confusing. We have worked together with experts to develop a tool that hopes to simplify these issues. Keep this protocol available to you, and your medical providers, in case of a medical emergency or admission.
Important steroid protocol topics covered:
- Corticosteroid conversions/equivalent doses
- Appropriate corticosteroid stress doses
- Recommendations for corticosteroid withdrawal
- Symptoms of adrenal crisis
- Tests that can help diagnose adrenal crisis
- De...
Source: Parent Project Muscular Dystrophy - March 2, 2015 Category: Neurology Source Type: news
PPMD & FED Award $1 Million Grant to ReveraGen BioPharma
Parent Project Muscular Dystrophy (PPMD) is excited to join forces with Foundation to Eradicate Duchenne (FED) to award ReveraGen BioPharma a $1 million grant to help fund the chronic toxicology program for VBP15, a combined action drug that addresses multiple levels of Duchenne. < div class="feedflare" > < a href="http://feeds.parentprojectmd.org/~ff/ppmd?a=f95iuOWULxw:TEkbujFIrf8:yIl2AUoC8zA" > < img src="http://feeds.feedburner.com/~ff/ppmd?d=yIl2AUoC8zA" border="0" > < /img > < /a > < a href="http://feeds.parentprojectmd.org/~ff/ppmd?a=f95iuOWULxw:TEkbujFIrf8:qj6IDK7rITs" > < img src="http://feeds.feedburner.com/~ff/pp...
Source: Parent Project Muscular Dystrophy - March 2, 2015 Category: Neurology Source Type: news
