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Summit to Extend Ongoing PhaseOut DMD Clinical Trial of Ezutromid in Patients with Duchenne Muscular Dystrophy

Summit Therapeutics announced today that it will proceed with the planned extension phase of PhaseOut DMD, a Phase 2 clinical trial evaluating the utrophin modulator ezutromid, subject to regulatory approval.

This follows an interim review of the safety and tolerability data from the ongoing trial by an independent Data…


Added by PPMD on March 27, 2017 at 11:25am — No Comments

Understanding Health Insurance Options: A Guide for People Living with Duchenne Muscular Dystrophy and their Caregivers

Health insurance is a contract between you and an insurance company. Health insurance helps pay your medical bills when you need care.

There are two major types of health insurance:

  • Private Health Insurance: Families and individuals obtain…

Added by PPMD on March 27, 2017 at 10:30am — 1 Comment

ReveraGen BioPharma Receives FDA Fast Track Designation for Vamorolone

Today ReveraGen BioPharma Inc, announced that the FDA has granted Fast Track designation for vamorolone (VBP15) for the treatment of Duchenne.  This designation can speed the review of efficacy and safety data for vamorolone in people with Duchenne, potentially leading to more rapid regulatory approval.…


Added by PPMD on March 24, 2017 at 12:30pm — No Comments

Community Update from PTC

Today, PTC Therapeutics provided the following update to the Duchenne community regarding their acquisition of Emflaza™ (deflazacort) from Marathon Pharmaceuticals. We appreciate PTC keeping our community updated during this transition.

March 24, 2017

Dear Duchenne…


Added by PPMD on March 24, 2017 at 10:38am — No Comments

Akashi Therapeutics Receives FDA Clearance to Resume HT-100 Clinical Development

PPMD is pleased to learn that the FDA has completed its review and concluded that Akashi Therapeutics may resume clinical development of HT-100 (delayed-release halofuginone) in people with Duchenne (click here to read Akashi's press release). Preventing fibrosis is an important target and an essential piece of a combination of therapies that will be…


Added by PPMD on March 22, 2017 at 9:00am — No Comments

PPMD Designates Ann & Robert H. Lurie Children's Hospital of Chicago a Certified Duchenne Care Center

Today Parent Project Muscular Dystrophy (PPMD) named Ann & Robert H. Lurie Children’s…


Added by PPMD on February 16, 2017 at 12:00pm — No Comments

PPMD’s #EverySingleOne Tour: Los Angeles, CA

Children’s Hospital Los Angeles (CHLA) marked our first stop of the New Year on PPMD’s 2017 Every Single One Tour, held on January 21, 2017. We were thrilled to not only be holding another tour stop at one of …


Added by PPMD on February 15, 2017 at 10:30am — No Comments

Marathon Pharmaceuticals Pauses Commercialization of EMFLAZA™ (deflazacort)

UPDATE: February 13, 2017, 5:10 PM -- An additional statement from Marathon has been added to the blog


Today, Marathon…


Added by PPMD on February 13, 2017 at 4:00pm — No Comments

PPMD to Host Forum on Access to Emerging Therapies on February 13th, 2017

Following decades of strategic advocacy, research investment, and biopharmaceutical development, the U.S. Duchenne community recently arrived in the post-approval space and is poised to receive additional novel therapies from a robust innovation pipeline.…


Added by PPMD on February 7, 2017 at 1:57pm — No Comments

PPMD Awards $600,000 Grant to NJIT & Talem for Next Phase of Upper Extremity Exoskeleton



Ensuring that the worlds of teens and adults with Duchenne 'grow bigger', as they grow older by expanding their reach – both literally and figuratively.


Combing your hair. Lifting a glass to your mouth. Washing your hands. Pressing elevator…


Added by PPMD on January 18, 2017 at 9:00am — 1 Comment

ATTENTION: All Patients on Masters’ Deflazacort

As you know, the FDA is currently reviewing Marathon Pharmaceutical’s New Drug Application (NDA) for deflazacort for the treatment of Duchenne. It…


Added by PPMD on January 16, 2017 at 6:30pm — No Comments

Akashi Therapeutics Provides Update on Three Duchenne Compounds

This weekend, Akashi Therapeutics announced they are working toward having three novel, complementary compounds in the clinic in 2017 with potential to treat all Duchenne patients independent of their specific genetic…


Added by PPMD on January 11, 2017 at 9:00am — No Comments

Sarepta Therapeutics Enters into Research Agreement and Option Agreement with Nationwide Children’s Hospital for Microdystrophin Gene Therapy Program

On the heels of PPMD’s announcement last week of a $2.2 million grant to Nationwide Children’s Hospital’s gene therapy study being led by Dr. Jerry Mendell and Dr. Louise Rodino-Klapac, Sarepta Therapeutics today announced their commitment to the trial through a separate research agreement with Nationwide Children’s, including an…


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

PTC Provides Update on Next Steps for Translarna

In a corporate update released today, PTC Therapeutics announced next steps for their drug, Translarna in the U.S. According to the release, PTC plans to file the Translarna New Drug Application (NDA) for nmDMD over protest with the U.S. FDA in…


Added by PPMD on January 9, 2017 at 10:30am — No Comments

Make my parents pay--48 hours left!

We’re so close to our goal to fund CRISPR/Cas9!…


Added by PPMD on December 29, 2016 at 9:30am — No Comments

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