PC Project - Home Page

2013 Patient Support Meeting - Santa Cruz, California

We now have over 30 PC Patients registered for the Santa Cruz patient support meeting. We encourage those who are planning to attend to register as soon as possible. More information is available on the events page.

RECYCLE & RAISE FUNDS FOR PC

Collect printer cartridges & small electronics in any used box. Once you have collected at least 20 items, ship them to Planet Green by printing a shipping label online.

Collect printer cartidges and small electronics in any used box. Once you have collected at least 20 items, go to http://www.planetgreenrecycle.com/pachyonychia and print a shipping label to ship the items for free.

Any of the following items can be sent and will raise money for PC research.

· Inkjet Cartridges

· iPods/MP3 Players

· Cell Phones /Pagers

· Digital /Video Cameras & Camcorders

· GPS & Radar Detectors

· PDAs

· Mobile Hot Spots

· iPads /Tablets

· Calculators

· Video Game Consoles & Handhelds

· eBook Readers

· Video Games & Accessories

Planet Green receives and inspects the items then they send PC Project a check.

Inherited Nail Disorders

Prof. Eli Sprecher gave a wonderful presentation at the August 2012 quarterly International Pachyonychia Congenita Consortium (IPCC) Physician Network meeting entitled Inherited Nail Disorders. The presentation goes through the reclassification, pathogenesis, and treatment of Pachyonychia Congenita and other nail disorders. The links are located on the Video & Presentations page from either the Patient or Physician/Scientist the drop down menu or just click here to view the quick time file. Feel free to comment on the blog and let us know what you think.

2012 Edinburgh Scotland Patient Support Meeting PSM Oct 28-30

There are now 14 PCers registered for the 28-30 Oct 2012 Edinburgh, Scotland Patient Support Meeting.

For those who are planning to go, please click here to register ASAP.
We would like to start to get a count of attendees.

Scholarships are available-if you need financial assistance to attend the Patient Support Meeting. Click here to apply for the scholarships online

Please contact PC Project if you have any questions or cannot register online.

LANDMARK RARE DISEASE LEGISLATION SIGNED INTO LAW BY THE PRESIDENT

President Obama signed into law yesterday the U.S. Food and Drug Administration (FDA) Safety and Innovation Act of 2012 which includes the most groundbreaking measures for rare disease patients and their families since the Orphan Drug Act of 1983. The Act will provide more than $6 billion in industry user fees to the FDA over the next five years to fund a share of the agency's review of drugs and medical devices. For rare disease patients, including pachyonychia congenita (PC) patients, it also includes provisions of critical importance. These come as a result of years of advocacy, education and relationship-building by a coalition of rare disease patient organizations, including PC Project. These provisions will accelerate rare disease patient access to new medical treatments, speed-up development of "breakthrough therapies", and improve consultation between the government and rare disease experts.

This legislation passed with bipartisan support in both the House and Senate. The FDA Commissioner, Margaret Hamburg, MD, as well told Congress that the FDA was committed to greater support of the rare disease program and closer collaboration with all those affected by rare disease research.

People with pachyonychia congenita, a rare disease affecting roughy several thousand world-wide, will benefit from this increased rare disease focus by the U.S. Government. PC Project's ongoing efforts to get cllinical trials underway for PC patients will be greatly assisted by this Act's provisions. We are thrilled to know we will be working in a governmental environment that will help us overcome regulatory and financial hurdles that are present in the drug development and approval process. We are grateful to everyone involved who worked on behalf of making these new rules a reality.

Pachyonychia Congenita featured in local news articles

We sent out a press release announcing that PC Project was selected to participate in the new Global Rare Disease Registry sponsored by the National Institutes of Health -- and the local newspaper and TV station contacted us to do a story. Click Here to read the interview with Janice Schwartz. If you'd like to have the press release to send to your local media outlets, please let us know and we'll be glad to share it with you. Perhaps you can generate a local story as well. That's great for advancing PC awareness. Thanks, Janice, for being willing to do the interviews. We know that's now always an easy thing to do.

LANDMARK RARE DISEASE LEGISLATION APPROVED BY CONGRESS!

Landmark legislation passed the U.S. Senate today aimed at helping to promote the development of rare disease therapies. Senate Bill 3187, the Food and Drug Administration Safety and Innovation Act of 2012 (FDASIA) will improve the FDA regulatory process, provide incentives to invest in rare disease therapies, and expand scientific capability to develop cutting-edge therapies. The House of Representatives approved the bill last week and it will now go to President Obama for his signature.

The bill's features important to rare disease patients will (1) enhance accelerated patient access to new medical treatments, (2) encourage the development of Humanitarian Use Devices, or medical devices for small patient populations, (3) provide accelerated development of "breakthrough therapies" - or ones that show early promise, (4) enhance consultation with rare disease medical experts, and (5) establish a rare pediatric disease priority review voucher incentive program.

PC Project has been working with the rare disease coalitions that have been providing input on, and supporting, this legislation.

PC Registry (IPCRR) featured in article on the importance of registries

This is a great article about the importance of registries -- and the example used is the PC Project registry (IPCRR). If you are not yet part of the IPCRR, please take time to share your information (fill out the Consent and Questionnaire) and take advantage of this great service. You will really be contributing to all that we hope to do for PC to find treatments and a cure. Those of you who are in thre registry -- take a bow! Dr. Irvine really explains how important you as a motivate patient community are in this important process.

PC Project Approved for Research Pilot Program

PC Project is one of 34 rare disease organizations approved to participate in a two-year pilot program of the Global Rare Disease Registry and Data Repository (GRDR) program launched by the federal government's Office of Rare Disease Research working in collaboration with Patient Crossroads, Children's Hospital of Philadelphia and Medscape/WebMD. The goal is to enable scientific analysis of data across many rare diseases and to facilitate clinical trials and other studies. We appreciate this increased scientific access this gives PC research. We look forward to sharing more details about this int he coming weeks. We are pleased to share our press release with you on this new and exciting opportunity!

Federal Grant for Scientific Meeting Awarded to PC Project

PC Project has been awarded a scientific meeting grant by the National Institutes of Health. This grant will support the upcoming meeting of the International PC Consortium to occur next February 2013 in Park City, Utah. This will be the 10th anniversary of this collaborative scientific group working on understanding PC and development of therapies. We are thrilled and honored. The NIH has been a great partner with PC offering support over the years for our scientific meetings. More details about the meeting will be provided soon. Any scientist wanting more information on this meeting is invited to contact PC Project.