Wow! What an unbelievable few weeks we have had in this community! Never in the 18 year history of PPMD has the Duchenne community been so ripe with promise and so full of hope. As I’m sure everyone is aware, Sarepta Therapeutics presented positive results from their 48-week results on eteplirsen, a drug designed to skip exon 51. PPMD will host a webinar for you to ask your questions, on October 23. Just last week, Summit announced that they have been able to achieve sufficient plasma levels of its reformulated utrophin-upregulating drug to justify a phase I/II trial in Duchenne. This is a therapy that would potentially help all people with Duchenne. PPMD has contributed to both therapies over the years and is excited for each company’s next steps. We have continued to fund important research by awarding grants to Dr. Dongsheng Duan for his gene therapy work in treating cardiomyopathy in Duchenne and Dr. Toshifumi Yokota at the University of Alberta who is experimenting with multiple exon skipping. This month’s World Muscle Society Congress in Perth, Australia promises one exciting announcement after another.
I also want you to meet a really cool guy: Wheelz. Wheelz (a/k/a Ryan Schmidt) is a rapper who, along with his family, hosts a huge Duchenne-awareness event in Austin every year. He uses music to enlighten audiences about Duchenne and is a force to be reckoned with. Meet Wheelz and make sure to download some of his music!
You will see that this month's newsletter is packed with information concerning advocacy, care, additional research news, incredible webinars, and the many goings-on in the community. It is a time like no other in this community and I hope, like PPMD, you will face the end of the year full of hope and optimism.
Where we are & what we're doing
PPMD makes it a priority to attend the most important meetings and conferences related to Duchenne, as well as connecting with the leaders in the field to move research, care, and advocacy forward.
See where we’ve been and where we are going:
Pat Furlong, Founding President, CEO
Read more PPMD Staff Blogs
Sign up to receive updates like this in your inbox every month.