Rare Disease Report

DMD Conference Begins This Week

JUNE 28, 2017
James Radke
The biggest Duchenne muscular dystrophy (DMD) meeting of the year begins this week, as the Parent Project Muscular Dystrophy (PPMD) will be hosting its Annual Connect Conference in Chicago from June 29 to July 3.
The event connects nearly 500 families from around the world to help them learn about how best to manage this disorder, and informs them of the plethora of treatment options currently in development.
Thursday’s agenda will include information for the newly diagnosed as well information for managing teens and young adults with DMD, and the afternoon will include 2 special speakers talking about the only 2 drugs currently approved for DMD – Ed Kaye, MD, of Sarepta Therapeutics and Stuart Peltz, PhD of PTC Therapeutics will talk about Exondys 51 (eteplirsen) and Emflaza (deflazacort), respectively.
On Friday and Saturday, investors and patients are likely to closely follow Twitter, as various pharmaceutical companies are expected to provide updates on their clinical trials.
Among the speakers scheduled to present:
  • Paolo Bettica, MD, PhD (Italfarmaco); Givinostat
  • Thomas Meier, PhD (Santhera); Raxone
  • Eric Hoffman, PhD (ReveraGen); Vamorolone
  • Linda Marban, PhD (Capricor); CAP-1002
  • Michael Binks, MD (Pfizer); domagrozumab
  • Cliff Bechtold, MS (BMS); anti-myostatin adnectin
  • Diana Escolar, MD (Akashi); HT-100
  • Joanne Donovan, MD, PhD (Catabasis); edasalonexent
  • Elias Kouchakji, MD (Fibrogen); pamrevlumab
  • George Mulligan, PhD (Mitobridge); MTB-101
  • Michelle Avery, PhD (Summit); ezutromid
  • Stuart Peltz, PhD (PTC Therapeutics); Translarna
  • Justin Fallon, PhD (Tivorsan); biglycan
  • Wendy Erler (WAVE Life Sciences); exon skipping
  • Paula Clemens, MD (NS Pharma); exon 53 NS-065/NCNP-01
  • Lisa Borland, PharmD (Sarepta); exon 45 and 53
Saturday afternoon will include special sessions on caring for DMD patients at various ages.
Sunday morning, the final few hours of the meeting, will include a special program involving robotics and other innovative ways that the DMD community is changing how disabled persons are staying involved in their daily lives.
For more information, about the meeting, visit PPMD's website. Also be sure to follow Rare Disease Report on Twitter for updates on the conference using the hashtag #PPMDConnect.

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