Parent Project Muscular Dystrophy Convenes Pulmonary Outcomes Workshop

Parent Project Muscular Dystrophy (PPMD) will convene a Pulmonary Outcomes Workshop in Bethesda, Maryland April 14-15. Leading physicians in the Duchenne pulmonary space, along with industry partners currently developing therapies that may impact the pulmonary dysfunction caused by Duchenne, as well as partners interested in developing future novel therapeutics, will gather to address a range of topics developed by the meeting's Steering Committee.

Duchenne muscular dystrophy is the most common fatal genetic disorder diagnosed in childhood, affecting approximately one in every 5,000 live male births. Changes in pulmonary function occur as Duchenne progresses. Respiratory muscles (including the diaphragm) weaken, making it more difficult for patients to breathe full breaths in, fully expand the lungs, hold full breaths, breath full breaths out, and to cough effectively. These changes and known to first occur during sleep and progress to also affect daytime respiration.

PPMD has a long history of exploring Duchenne, both from an organ-specific perspective, as well as systemically, throughout the lifespan. Currently, there are several measures used to demonstrate changes in Duchenne pulmonary function.  PPMD believes it is time to take a deeper dive into the evaluation of these measures, which include:

  • Changes in inspiratory strength:  Strength of the muscles while breathing in/ MIP-maximal inspiratory pressure.
  • Changes in expiratory strength: Strength of muscles while breathing out/ MEP- maximal expiratory pressure, PEP - peak expiratory pressure.
  • Changes in the volume of air that the lungs can hold: FVC or forced vital capacity; FVC% - forced vital capacity when compared with normal; PEF - peak expiratory flow.

Clinical trials that hope to improve pulmonary function include many of these measures of pulmonary function as pulmonary outcome measures. But using pulmonary outcome measures creates a whole series of questions needing clarification.

As such, PPMD convened an expert Steering Committee to face these questions head on. Thanks in part to support from Santhera Pharmaceuticals, Catabasis Pharmaceuticals, and Pfizer Inc., PPMD will gather experts in Duchenne pulmonary care and evaluation, to discuss these questions.

The goals of this Pulmonary Outcomes Workshop include:

  • Assess currently utilized pulmonary outcome measures, their clinical meaningfulness, possible limitations, and the need to implement novel pulmonary outcome measures
  • Identify how our current knowledge of pulmonary outcome measures can further drug development
  • Identify gaps in our current knowledge of pulmonary outcome measures
  • Utilize this forum to educate ourselves, the greater Duchenne community, and the FDA on the need for reliable, responsive, and meaningful pulmonary outcome measures

In order to achieve these outcomes, we will discuss:

  • The natural history of pulmonary function data demonstrated in several natural history data sets
  • The correlation of pulmonary function with skeletal muscle strength and function, incidence of upper respiratory infections/pneumonia/antibiotic use
  • The analysis of pulmonary data, and whether data sets can be compared
  • Whether the implementations of standardized protocols might enhance the reliability of pulmonary outcome measures
  • Novel assessments that might be implemented in future trials  

PPMD has invited members of the FDA Divisions of Neurology and Pulmonology to participate in this workshop.

Kathi Kinnett, MSN, CNP, PPMD's Senior Vice President of Clinical Care is excited to bring together this incredible group of experts and thinks the meeting will be invaluable:

"It is our hope that this workshop will help to clarify much of the confusion around pulmonary outcome measures.  We must begin by agreeing on which pulmonary outcome measures to collect, how to analyze these measures, and the meaningfulness of these measures. If we can agree on these issues, we can begin to demonstrate the efficacy of drugs on pulmonary function and, hopefully, speed up the drug approval process. It's a lofty goal but, with this panel of experts, we believe we are up to the task."

PPMD plans to publish results from the workshop later this year. For more about PPMD's extensive approach to Duchenne care, please visit our website. 

Thank You to Our Partners for Supporting PPMD's Pulmonary Outcomes Workshop:


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