Matt’s Promise is dedicated to making a difference
in the lives of young people affected by terminal illnesses.
Our cornerstone project is to find a cure for Duchenne Muscular Dystrophy (DMD).

Matt’s Promise is dedicated to making a difference in the lives of young people affected by terminal illnesses.
Our cornerstone project is to find a cure for Duchenne Muscular Dystrophy (DMD).

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Highlights from 12th Annual Benefit Concert

Thank you to all of our supporters!

Enjoy the photos from the Matt’s Promise 12th Annual Benefit Concert.

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Race to Yes Urges FDA to Approve Eteplirsen to Treat Duchenne Muscular Dystrophy

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The Race to Yes initiative is urging the U.S. Food and Drug Administration (FDA) to carefully consider the evidence presented at the latest Advisory Committee meeting and to approve fast access for the Duchenne muscular dystrophy drug, eteplirsen.

“Monday’s advisory committee meeting on eteplirsen saw a unified show of support from the patients and scientists who live with, study and treat Duchenne. The testimony from Duchenne experts during the open public hearing clearly underscores the positive data generated in the small clinical trial,” the co-founder of Race to Yes, Tracy Seckler of Charley’s Fund, said in a press release.

The Advisory Committee to the FDA, an independent panel of experts, met on April 25 to review eteplirsen. During the 12-hour meeting attended by more than 800 researchers, clinicians, and patients and their families from all over the world, compelling evidence in support of the efficacy and safety of the drug were presented.

However, the advisory committee voted against accelerated approval based on the fact that eteplirsen’s effect was too small and variable.

In clinical trials conducted over five years, eteplirsen has been shown to delay the progression of the disease based on the six-minute walk test, which measures the distance walked by trial participants in six minutes. But the trial only involved 12 boys.

The FDA will take into account the recommendations of the advisory committee, but is not obliged to follow them.

Under Food and Drug Administration Safety and Innovation Act (FDASIA), Congress gave the FDA the authority to grant accelerated approval for new treatments for life-threatening rare diseases such as Duchenne muscular dystrophy (DMD).

Marissa Penrod of Team Joseph, a co-founder of Race to Yes, said: “We encourage the FDA to honor FDASIA, and fully utilize the tools Congress has given them.”

A final FDA decision on whether to grant accelerated approval for eteplirsen, which is produced by Sarepta Therapeutics, is expected on May 26.

Eteplirsen is a drug designed to restore the reading frame of the dystrophin gene. In some Duchenne cases, a portion of this gene is missing, resulting in the remaining part not “fitting together” properly. By skipping over part of the remaining gene, the reading frame can be restored allowing the body to make a shorter but still functional dystrophin protein.

Race to Yes is a community-driven initiative that supports and campaigns for the rapid regulatory approval of DMD drugs.

Originally published at musculardystrophynews.com

‘Hunger Games’ stars scheme with teen on prank to support Duchenne muscular dystrophy research at UCLA

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“Hunger Games” stars Jennifer Lawrence, Liam Hemsworth and Josh Hutcherson teamed with 14-year-old Dylan Miceli-Nelson to prank the stars of SMOSH, Anthony Padilla and Ian Hecox, to raise money for Duchenne muscular dystrophy.

A local teenager living with Duchenne muscular dystrophy will attend tonight’s premiere of “The Hunger Games: Mockingjay, Part 2” — and the backstory couldn’t be more Hollywood.

Thanks to the help of “Hunger Games” stars Jennifer Lawrence, Liam Hemsworth and Josh Hutcherson and the practical-jokes-for-good-causes website “Prank It FWD,” 14-year-old Dylan Miceli-Nelson’s appearance at the star-studded premiere is also a rare opportunity to raise awareness and funds for the Center for Duchenne Muscular Dystrophy at UCLA, which is led by Dylan’s parents, Stan Nelson and Carrie Miceli.

(more…)

Eteplirsen For Duchenne Muscular Dystrophy Should Receive FDA Accelerated Approval

About: Sarepta Therapeutics, Inc. (SRPT), Includes: BMRN

Disclosure: I am/we are long SRPT.

Summary

Clinical studies have demonstrated biochemical efficacy and safety of Eteplirsen.

Clinical benefits of Eteplirsen have been shown in a small patient group, and are likely to be reproduced in larger trials.

Eteplirsen is poised to receive FDA accelerated approval and serves as an attractive short-term investment.

Eteplirsen is an experimental antisense drug for the treatment of a rare and fatal disease called Duchenne muscular dystrophy (NYSE:DMD). It is developed by Sarepta Therapeutics (NASDAQ:SRPT) as the leading product in the company’s pipeline. The U.S. Food and Drug Administration (FDA) has accepted the New Drug Application (NDA) of Eteplirsen through the accelerated approval program. An advisory committee meeting is scheduled for January 22, 2016, with a Prescription Drug User Fee Act (PDUFA) date of February 26, 2016.

Akashi Reports Positive Clinical Data on HT-100

Akashi Therapeutics Reports Positive Clinical Data on HT-100 in Patients with Duchenne Muscular Dystrophy

Download as PDF

Interim Clinical Data from Ongoing Phase 1b/2a Clinical Program Highlights Statistically Significant Improvements in Muscle Strength

Cambridge, Mass. – June 18 , 2015 – Akashi Therapeutics Inc., a clinical stage biopharmaceutical company developing treatments for Duchenne muscular dystrophy (DMD), today announced positive interim clinical data from an ongoing Phase 1b/2a clinical program with HT-100 (delayed-release halofuginone) an orally available, small molecule developed to reduce fibrosis and inflammation and promote healthy muscle fiber regeneration in boys with DMD. (more…)

One year later: An important infographic update

One year ago, you helped us rally more than 106,000 people to sign a White House petition urging approval for safe and effective treatments for Duchenne. Today, a full year later, we are still fighting for the fast and efficient regulatory approach that our children deserve.The FDA’s actions make it clear that they do not share our urgency. We have more work to do to get them on the right track. We will not let up until all kids with Duchenne have access to safe and effective treatments, and we hope you are still with us to help.Let’s demand accountability from the FDA. Let’s tell Congress ENOUGH IS ENOUGH. Let’s contact FDA Commissioner Hamburg on her way out the door and the new Commissioner on his way in so the FDA knows that Duchenne is an urgent priority, that we are fully aware of their flip flopping and indecision, that we are watching them closely, and that ENOUGH IS ENOUGH.

PLEASE TAKE ACTION NOW

The first step is simple: Share this infographic on Facebook, Twitter, Instagram, and everywhere else with @US_FDA and the hashtag #getbackontrack and #racetoyes View the full graphic

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Meet Akashi Therapeutics

Nothing’s more frustrating than shelved potential. But in 2010, Dr. Benjy Seckler read about a compound showing promise in animal models of Duchenne. The therapy, initially created for a different disease, hit a dead-end when tested in humans: it triggered nausea and vomiting. This and other roadblocks proved too daunting for the Israeli biotech that owned the asset, and development was halted.

0001Knowing this compound had potential to benefit children with Duchenne, Dr. Seckler would not take no for an answer. He had to figure out a way to take the molecule, address the side effects, and get it into a clinical trial for kids with Duchenne. The solution turned out to be creating a new biotech company, which was called DART Therapeutics.

Fast-forward 3 years. The drug is now called HT-100 (affectionately referred to as “Halo” because its main ingredient is halofuginone), and is presently completing a Phase 1B/2a trial in which Charley is participating. Early data was released this summer at a scientific conference, and it is encouraging. If DART could do this for Halo, why not for other therapies? (more…)