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All Blog Posts Tagged 'PPMD Blog' (713)

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

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

ACTION ALERT: Make Your Voice Heard on New Healthcare Proposal

“Who’s on first?”

Remember that classic Abbott & Costello routine? Well, trying to track the quickly-shifting healthcare bill has felt much like watching that routine. Complex, confusing, and exhausting. But the stakes here are no laughing matter.




Added by Annie Kennedy on March 21, 2017 at 10:00am — No Comments

ENMC Workshop Report: Airway Clearance Techniques in Neuromuscular Disorders

The European Neuromuscular Center (ENMC) is a global organization that gathers expertise from around the world to address many issues and concerns of, and advance research and treatments for, neuromuscular…


Added by Kathi Kinnett on March 17, 2017 at 4:00pm — No Comments

PPMD's Duchenne Drug Development Roundtable Meeting Series, Part 2: Optimizing Clinical Trials

This week, PPMD convened Duchenne industry partners, experts in adaptive trial design and biostatistics, and innovative partners from outside of the Duchenne world who’ve revolutionized their clinical trial spaces for the second meeting of our Duchenne Drug Development Roundtable's 3-part meeting series, focusing on Clinical…


Added by Abby Bronson on March 3, 2017 at 10:00am — No Comments

PPMD Joins Rare Disease Advocates in Letter to President

PPMD joined 200 patient organizations in a letter to President…


Added by Annie Kennedy on February 27, 2017 at 7:30pm — No Comments

Duchenne Research Update: February 2017


An Overview of Your Impact in Action

PPMD Grant Awards in the last Quarter of 2016 and first part of 2017.


PPMD Launches Long-Term…


Added by Abby Bronson on February 24, 2017 at 1:29pm — 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

Deflazacort Approved in the U.S. for Duchenne

UPDATE 2/10: Marathon has released links to new resources, including the EMFLAZA website (https://emflaza.com) and a Patient Support FAQ document (…


Added by Pat Furlong on February 9, 2017 at 5:00pm — 2 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’s Duchenne Drug Development Roundtable: Bringing Industry Together to Accelerate Research

Better. Faster. Now.


How do we apply the ‘regulatory learnings’ of the past 18 months back into our robust Duchenne pipeline to ensure as many effective therapies yield from our pipeline as possible?

Under the leadership of PPMD's Duchenne Drug Development Roundtable (DDDR), our community is determined to figure that out. Today is the first of a…


Added by Abby Bronson on February 1, 2017 at 10:00am — No Comments

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