Work continues on the part of 237 patient advocacy organizations to try to pass the OPEN Act (Orphan Product Extensions Now, Accelerating Cures and Treatments; HR 1223 / S 1509). The HPS Network is one of the patient organizations supporting this bill.
The OPEN Act offers incentives to pharmaceutical companies to repurpose existing drugs for use to treat rare diseases. Currently, only 5 percent of the more than 7,000 rare diseases have an FDA approved treatment. Many symptoms for rare diseases, like HPS, are often treated with medications that are off label, and thus can sometimes be difficult to pay for. The bill would offer pharma companies an extended six months of patent exclusivity if they work to repurpose medications for use in rare diseases. The sponsor company must demonstrate that the repurposed therapy is designated to treat a rare disease and obtains an approved rare disease indication from the FDA on the drug label. Repurposing drugs is faster, cheaper, and presents fewer risks than traditional drug development.
The legislation has the potential to double the number of treatments available for rare disorders.
Asking your House and Senate representatives to support, or co-sponsor, this legislation is easy. Go to this link and it will automatically locate your representatives as well as provide a ready-made letter you can send them. Just enter your information in the provided fields. http://action.everylifefoundation.org/p/dia/action4/common/public/?action_KEY=22781
The second annual A Run Through History will be held Sunday, Oct. 22nd at 8:30 am at Theodore Roosevelt Memorial Park in Oyster Bay, NY. The event will benefit the HPS Network and PeerPals.org. To participate in the race, there is a $25 entrance fee, plus a $2.50 sign-up fee. Come run, or just walk, with your friends!
The HPS Network is an Oyster Bay-based non-profit serving those impacted by Hermansky-Pudlak Syndrome or HPS. HPS is a type of albinism that causes decreased visual acuity (often legal blindness), a bleeding disorder, and in some types bowel and lung disease.
Peerpals.org is also an Oyster Bay-based non-profit that provides play dates and friendship opportunities between preschool age kids with disabilities and their non-disabled peers in the community.
For more information, go to www.arunthroughhistory.org
Joanne Criblez’s summer camp voice students are giving a concert to raise money and awareness for Hermansky-Pudlak Syndrome (HPS) Thursday, Aug. 10th at the LIU-Post Hillwood Commons Theatre, 720 Northern Blvd., Brookville, NY. The performance is “A Night At The Movies Summer Cabaret” and will feature music from iconic film scores.
Tickets are $10 and available at the door.
Joanne Criblez is a long-time supporter of the HPS Network and Ashley Appell’s voice coach. For many years she organized the HPS Christmas Concert with the J-Band.
The HPS Network is very grateful to her, and all of the campers who will be singing on our behalf. We thank you from the bottom of our lungs!
In July, the HPS Network received a check for $86.56 from www.igive.com – an online shopping mall that allows merchants to donate a percentage of your purchase price to the cause of your choice. Granted, this is not a ton of money, but it will pay for six new patient packets. Everything matters because the HPS Network is such a small non-profit. Thanks for shopping at www.igive.com and please, keep it up! Share it with friends, and ask them to choose the Hermansky-Pudlak Syndrome Network as their charity.
Some of the stores you’ll find at iGive include:
- Verizon (If you go to the Verizon website to pay your bill online, Verizon will donate a percentage of your bill to the HPS Network.)
- Eddie Bauer
- JC Penny
- And, more than 700 other retailers
Oyster Bay, NY – A study location for an observational study of Hermansky-Pudlak Syndrome has opened at Loyola University in the Chicago area. Hermansky-Pudlak Syndrome, or HPS, is a type of albinism that involves other health-related issues beyond low vision and pigmentation such as a bleeding disorder, and in some gene types, bowel and lung issues. The lung disease of HPS is called Pulmonary Fibrosis and causes a scarring of the lungs that, without a lung transplant, is ultimately fatal. HPS occurs around the world, but is most common in people who trace their family roots to Puerto Rico.
This observational study will help researchers identify the earliest measurable pulmonary disease activity in people affected by HPS. It also hopes to identify biomarkers that will aid in the understanding of the cause of pulmonary fibrosis in those with HPS types 1, 2 and 4. This could help facilitate more rapid conduct of therapeutic trials in HPS for patients with early lung disease in the future.
The study is part of the Rare Lung Disease Consortium and is co-sponsored by Vanderbilt University, the University of Florida, the National Institutes of Health (NIH) and the National Heart, Lung and Blood Institute (NHLBI).
Eligible people are those with HPS, ages 12 to 90, with any HPS gene type. The observational tests could include a blood draw, urine samples, breathing tests and a CT scan.
To learn more about the study, or how you might participate, contact the HPS Network, (516) 922-4022 or e-mail: firstname.lastname@example.org.
The National Organization for Albinism and Hypopigmentation (NOAH) is sponsoring a scholarship to help with registration fees for the Space Camp for Interested Visually Impaired Students (SCIVIS) at the U.S. Space and Rocket Center in Huntsville, AL. The scholarship only assists with the camp registration fees. Transportation and other expenses are up to the camper’s family to cover. The deadline to apply is rapidly approaching. To apply, find out more at: http://www.albinism.org/site/c.flKYIdOUIhJ4H/b.9260627/k.B03/Programs__Services__Space_Camp_Scholarship.htm
Donna Appell, Executive Director and Founder of the HPS Network, has been nominated for the Eagle Rare Life Award, a contest held by the Eagle Rare Bourbon company. To win, however, she needs your vote every day until Dec. 5th. If Donna wins, the prize is $50,000. There are also six runner-up awards of $5,000 each. Voting can get us into the top 30 finalists, but after that, the votes are 20 percent of the score. You can only vote every 24 hours. It just takes a second! Go to: http://eaglerarelife.com/content/donna-appell
The Boxcar Bar and Arcade in Greensboro, N.C. held an Fundraiser Night on June 28 to benefit the HPS Network. They donated 15 percent of sales to the HPS Network from the night. Karen and Tommy Tillman worked with Boxcar on the event and offered information about the HPS Network that night. Boxcar Bar and Arcade is a bar that also offers an array of video game classics the way you might remember them from the video game arcade days, from Pacman to Pinball. Attendees spent the evening playing games and having fun while raising more than $400 for the HPS Network!
Heather Kirkwood, VP and Director of Communications for the HPS Network, represented the HPS patient community at the first NCATS Day at the National Institutes of Health (NIH). NCATS, or the National Center for Advancing Translational Science, is one of 27 institutes and centers at the National Institutes of Health (NIH). It is charged with transforming the translational process so that new treatments and cures for disease can be delivered to patients sooner.
Each section of NCATS gave a short presentation on its function, and then attendees were divided into six groups to discuss how NCATS can better, and most efficiently, interact with patient communities to ensure the patient voice is included in the research process.
“I was really impressed with the number of NCATS staff that attended the event, and how truly excited they seemed to be interacting with the patient community,” said Kirkwood, “I’m really looking forward to participating in future NCATS events and watching this process unfold further.”
Jean-Marc D. Quach, President and CEO of the Alpha I Project Inc., and Michael Iannotti, PhD and Postdoctoral Fellow with the Division of Pre-Clinical Innovation at NCATS gave a joint presentation on how the Alpha 1 community and NCATS have partnered on Alpha 1 research. “It was a great presentation. Coming from a journalism background, I love case studies and look forward to seeing more of them representing all type of patient groups of all sizes and resource levels,” said Kirkwood.
Much of the day was then spent between NCATS staff and community representatives to exchange ideas and provide feedback.
The documentary RARE, which followed the HPS Network through the process of a clinical trial during a three-year period, is available for DVD purchase. Be sure to use this link that takes you to the home video section of the distributor’s website. It is sold elsewhere on the site to institutions or organizations that will screen the film publicly and is more expensive there. These copies are intended for home viewing. http://homevideo.icarusfilms.com/fanlight/rare.html