Getting things done

If you're following us on facebook (and I hope you are: https://www.facebook.com/xlhnetwork), you probably saw a picture of Network president, Becky Mock, along with Board members Oliver Gardiner (who flew all the way over to North Carolina from London!) and Geoff Edelson (plus other member volunteers, Rebecca Putnam, Meredith Mock and Sheila Hunter) at the ISPE-CaSA conference.

The conference is an annual gathering of technical professionals in the pharmaceutical and medical device manufacturing industries. It was an opportunity to interact with a different group of medical professionals than we've previously met.

As Becky told me, they "had a great time, made new friends for the Network, received a five thousand dollar contribution from the organization and raised an additional thousand dollars in a raffle."

It was also an opportunity for board members to meet with representatives from Ultragenyx Pharmaceutical, including Dr. Ali Skrinar (senior director, clinical sciences) and Kim Mooney (Associate Director of Patient Advocacy).

Ultragenyx describes itself as "a clinical-stage biotechnology company committed to bringing to market novel products for the treatment of rare and ultra-rare diseases." Of particular interest to Network members, Ultragenyx is collaborating on the development and commercialization of KRN23 for treatment of XLH. Further information about KRN23 is expected to be released during an upcoming global conference for endocrinologists in Chicago on June 21-24. We'll have updates here as soon as we can.

In addition to the meetings and money (both of which are nice, of course), the ISPE-CaSA event also served as a deadline for completing some tasks that have long been on the Network's agenda. You may remember the video that SAB member, Carolyn Macica, was instrumental in putting together. We also updated our brochure, to include new research and other useful information. Finally, we had a banner made, so people could find us at the ISPE-CaSA event (and in future events where we have a booth).

XLH Network members raise over $5,000

The Inmans, whose daughter has XLH, held a fundraiser this past Spring raising over $5000 for the XLH Network.   The fundraiser engaged local business owners, friends, and family and included raffle items, proceeds from dinner receipts, and sales of t-shirts designed by their daughter.   

In addition to raising money, the event raised awareness for XLH and showed that a small grass roots effort from dedicated people can have a huge impact.  

Below is a picture of Sandesr Inman presenting her t-shirt to Dr. Carpenter at the Yale Clinic for XLH.