June 2014 Blog Posts (17)

PPMD Welcomes John Porter!

John Porter, PhD is a Program Director at the NIH/NINDS, responsible for managing a portfolio of research grants across neuromuscular disorders. That is his formal introduction, but the other side is that he has long been a friend to me and to our Duchenne community. In January, he announced his intention to retire from NIH. RETIRE? JOHN PORTER? What’s next I wondered?…


Added by Pat Furlong on June 28, 2014 at 5:43pm — No Comments

Muscular Dystrophy Organization Nepal

Muscular Dystrophy Organization Nepal

Banepa  Kavre

Muscular Dystrophy Organization Nepal is dedicated for the rehabilitation approach for the muscular dystrophy adults, children to provide them 24 hours caring services, to provide them food, clothes, medicines, physiotherapy, regular health check- up, skill development programs like paintings, computers, a lots of skills programs, educate parents ,communities about the disease with the associate with the…


Added by Nirmal Khadka on June 27, 2014 at 2:59pm — No Comments

Strength Happens Together: PPMD Submits FDA Draft Guidance on Duchenne

June, 25, 2014 – Today is a landmark day for PPMD and the entire Duchenne community. We are so pleased that at our organization’s 20 year anniversary – after 2 decades of hard work and resolute persistence within the policy, legislative and regulatory arena – we are today able to provide to the US Food and Drug Administration the…


Added by Pat Furlong on June 25, 2014 at 11:00am — No Comments

Illinois Designates Week of June 23 as Duchenne Awareness Week!

The State of Illinois is recognizing our 20th Annual Connect Conference in Chicago, IllinoisJune 26-29, 2014 by declaring the week of June 23 as Duchenne Muscular Dystrophy Awareness Week! With over 500 families and Duchenne experts expected…


Added by PPMD on June 23, 2014 at 1:30pm — No Comments

PPMD Convenes Outcome Measures Meeting

PPMD will convene a pre-conference meeting of physical therapists, occupational therapists, and clinical coordinators who specialize in the research, assessment, longitudinal data collection, and treatment of people with Duchenne, June 25 and 26 in Chicago. This meeting will precede…


Added by PPMD on June 19, 2014 at 2:30pm — No Comments

Progress Update! House Subcommitee Clears MD-CARE Act for Next Step

UPDATE - June 20, 2014

Progress! Yesterday the House Energy & Commerce Subcommittee on Health voted via unanimous voice vote to advance H.R. 594, the Paul D. Wellstone Muscular Dystrophy Community Assistance, Research and Education Amendments of 2014. This action moves the bill one step…


Added by Ryan Fischer on June 18, 2014 at 9:30am — No Comments

Parent Project Muscular Dystrophy Names Children's Hospital Colorado a Certified Duchenne Care Center

Children's Hospital Colorado (Children's Colorado) was named a Certified Duchenne Care Center by Parent Project Muscular Dystrophy (PPMD), the leading advocacy organization working to end Duchenne muscular dystrophy (Duchenne).

Duchenne is the most…


Added by PPMD on June 17, 2014 at 12:32pm — No Comments

206th ENMC Workshop on Adults with Duchenne

We don’t know a lot about adults living with Duchenne: their natural history, cardiac progression on and off steroids, on an off cardiac medications, the effects of other medications and/or supplements, endocrine issues, gastrointestinal issues, urologic issues, gaining and maintaining independence……..the list could go on and on. Who would have dreamed that one day we…


Added by Kathi Kinnett on June 16, 2014 at 1:06am — No Comments

Pulmonary Care and Duchenne

Recently, Dr. Lisa Wolf (Assistant Professor, Pulmonary Medicine and Pulmonary and Neurology at Northwestern University, Feinberg School of Medicine, Chicago, IL) gave a wonderful presentation at Duchenne Foundation Australia’s Melbourne Symposium. While her presentation was titled, “DMD Respiratory Review 2014: The Younger Years,” she…


Added by Kathi Kinnett on June 16, 2014 at 12:34am — No Comments

Healthy Summer Snacking & Activity

It’s summer – finally! Growing children are hungry children, and hungry children don’t usually go for the healthy snacks first.

As parents, we try everything we can to get them to eat right: reasoning (this sometimes works), bribery (generally referred to as “creative parenting”) and sneaking nutrients into the foods they love (referred to as,…


Added by Kathi Kinnett on June 16, 2014 at 12:30am — No Comments

"Right to Try" State Model Legislation

The Goldwater Institute of Arizona has designed state model legislation, known as a "Right to Try" bill, which would make experimental drugs available to terminally-ill patients without the FDA's approval. Under this legislation, a patient with "an advanced stage of a disease with an unfavorable prognosis and no known cure" could obtain an experimental drug that has passed…


Added by PPMD on June 16, 2014 at 12:00am — 2 Comments

How we are doing.

Wow, it has been forever since I connected with families on PPMD.  Life has been crazy busy for our family.  Wyatt started Kindergarten year, which has been a huge adjustment.  We just got back from Cincinnati and all his well.  HIs motor skills are stable and heart is great.  Blessings!

Added by Wyatt's Mommy, Melissa on June 14, 2014 at 5:52pm — No Comments

Clinical Development of HT-100 for Duchenne Has Resumed

Phase 1b/2a Clinical Program Resumes Enrollment and Dosing in DMD Patients 

Preliminary Clinical Data to be presented at Upcoming Medical Conference 

Read the news release from DART Therapeutics:

DART Therapeutics Continues Clinical Development of HT-100 for Duchenne Muscular Dystrophy 

Cambridge, Mass.—June 12,…


Added by PPMD on June 12, 2014 at 12:23pm — 5 Comments

PPMD Benefit/Risk Study Results Published in Clinical Therapeutics

PPMD is thrilled that results from the recent benefit/risk project our team led have been published in Clinical Therapeutics. The article concludes that caregivers are willing to accept a serious risk when balanced with noncurative slowing or stopping of the progression…


Added by Holly Peay on June 9, 2014 at 1:30pm — No Comments

Lend Your Voice to the FDA Draft Guidance on Duchenne to Speed the Development of Therapies

Strength Happens Together

For the past four months, over 80 experts in Duchenne (including patient representatives and groups, industry, clinicians, and researchers) have been working…


Added by Ryan Fischer on June 4, 2014 at 4:00pm — No Comments

Prosensa Announces A Regulatory Path Forward for Drisapersen as a Potential Treatment for Duchenne

Prosensa Plans to Submit a New Drug Application to the FDA this Year; Dialogue with EMA continues with intent to seek approval

More good news from the FDA! This news from Prosensa continues to demonstrate that FDA recognizes the urgent needs of this community, the importance of the patient voice to understand the benefit/risk assessment, and continues to…


Added by PPMD on June 3, 2014 at 9:00am — No Comments

A New Journey

A New Journey

I had often wondered on my journey where God would take me. As a young lady I had dreamed of falling in love, getting married and having a family...like any female would. But as life would have it, things didn't go according to my plan, of course. Things went a whole different direction. A direction that maybe would be full of surprises. A lot of good and then some bad. Either way it would be a journey directed by God.

Once my son Tim was…


Added by Dee on June 2, 2014 at 12:30pm — No Comments

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