2386 East Heritage Way, Suite B

Salt Lake City, UT 84109

877-628-7300

www.pachyonychia.org

THANK YOU for participating in the first Pachyonychia

Congenita Golf Tournament. All donations to PC Project are

gratefully received and are matched $2-for-$1 and donated

funds are used 100% to fund research and support services.

Gold Sponsor — Mantyla McReynolds, CPA

Silver Sponsor — Zions Bank

Hole-In-One — Nate Wade Subaru

Saturday, August 20, 2011

The Homestead Golf Resort

Midway, Utah

TOURNAMENT SPONSORS

801-269-1818

Matt Johnson

801-844-7867

(801) 258-0590

HOLE SPONSORS

Jeff Pederson

801-562-3001

Jeremy Jackson Brandon LeRoy

(801) 277-3927

Buca di Beppo

Brigham Young University

Café Rio

Citris Grill

Corner Bakery Café

Five Guys Burgers & Fries

Massage Envy

Outback Steakhouse

Pei Wei Asian Diner

The Cheesecake Factory

Uinta Golfa

Cinemark Movie Theater

University of Utah

Utah Grizzlies

Utah Jazz

Raffle, Silent Auction and Prize Donors

David Frey Trent JefferiesDavid Frey Trent Jefferies 801801--274274--20452045

801-336-0564

Utah Cribs Real Estate Team Brad Miles - (801) 428-7223 Jake Breen, Spencer Janke

Wendy Wheeler 801-550-5350

1-888-346-0990

Corporate and Individual Health Programs

TEEBOX SPONSORS

801-328-3131

Jim Anderson

(801) 860-6200

801-943-5111

BOARD OF TRUSTEES

David B. Barlow (CEO)

C. David Hansen, MD (Dermatologist)

Roger L. Kaspar, PhD (Scientist and CEO)

F. Dwight Marchant (Bank Executive; retired)

Lex L. Udy. PhD (Scientist and CEO; retired)

Craig T. Vincent, Esq. (Attorney at Law)

MEDICAL AND SCIENTIFIC ADVISORY BOARD

Sherri Bale, PhD—GeneDx,

C. David Hansen, MD—University of UT, Dermatology

Peter R. Hull, MD—University of Saskatoon, Dermatology

Roger L. Kaspar, PhD—TransDerm,Inc, Santa Cruz, CA

E. Birgitte Lane, PhD—

W.H. Irwin McLean, DSc, FRSE—University of Dundee, Scotland

Leonard M. Milstone, MD—Yale University, Dermatology

Edel O'Toole, MD, PhD—Barts & the London, Queen Mary’s, Dermatology

Amy Paller, MD—Northwestern University, Dermatology

Dennis R. Roop, PhD—University of CO, Gates Stem Cell Center

Frances J.D. Smith, PhD—University of Dundee, Scotland

Eli Sprecher, MD, PhD—Tel Aviv Sourasky Medical Center, Dermatology

Maurice van Steensel, MD, PhD—University Maastricht, Dermatology

Jean Y. Tang, MD, PhD—Stanford University, Dermatology

PC Project Staff

Holly Evans, Administrative Assistant

Janice Schwartz, Patient Advocate (volunteer)

Billy Oslund (volunteer)

Mary Schwartz, Director (volunteer)

PC PROJECT CHARITY GOLF TOURNAMENT 2011 PC PROJECT CHARITY GOLF TOURNAMENT 2011

Hole # Sponsor Signs Contest Signs Prize

1 Mantyla MCREYNOLDS Photographs

2 Hole-in-one PING irons

Closest to the hole BYU “Stuff” Bag

3 Jackson & LeRoy Remodeling

4

5 Stoel-Rives

6 VendRx

7 Zions Wealth Advisors Long Drive PING putter

8 Nate Wade Subaru Hole-in-one New Car $26,000

9 Utah Cribs Real Estate

10

11 Kirkham & Friends Real Estate

12 Hole-in-one $500 cash

Closest to the hole Planet Fitness

13 Orriant

14 Hole-in-one Apple iPad

Closest to the hole Planet Fitness

15 Just4You Window Cleaning

16

17 Zions Wealth Advisors Long Drive TaylorMade putter 18 Del Sol & CariLoha

Tournament Contests and Prizes

RAFFLE—4 rounds of golf and over 40 other items totaling more than $1,400.00

in prizes — and don’t miss your chance to bid on the Silent Auction items.

1st Place Team—Four $60.00 Certificates at the Homestead Pro Shop and Four DelSol Sunglasses

2nd Place Team—Four $40.00 Certificates at the Homstead Pro Shop and CaRiLOHa tee shirts

3rd Place Team— Four CaRiLOHa golf shirts and four DelSol Frisbees

Plus High Score...Women’s Teams...and more

In December 2003, only three

patients were registered with

PC Project.

There are now over

1000 patients in more than 50

countries — and almost half have

received their genetic testing and are

participating in the International PC

Research Registry.

PC PROJECT CHARITY GOLF TOURNAMENT 2011

What is Pachyonychia Congenita? PC is an ultra rare genetic skin disorder caused by a single

mutation in one of at least four keratin genes including K6a, K6b, K16 or K17. PC may be he-

reditary (inherited from a parent who has PC) or may be spontaneous (a mutation occuring

when no parent or other family member has PC). Features of PC may include:

1) Painful blisters and calluses on hands and feet (focal palmar and plantar hyperkeratosis)

2) Thickened Nails (hypertrophic nail dystrophy or pachy-onychia)

3) Follicular hyperkeratosis (bumps around hairs at sites such as waist, hips, knees, elbows)

4) Leukokeratosis of the oral mucosa (white film on tongue, cheeks and sometimes larynx)

5) Cysts of various types (including steatocystoma and pilosebaceous cysts)

Change this... to this

Help us deliver a treatment and a cure — the gene therapy

research will also benefit many other disorders.

All Donations Welcome

Visit www.pachyonychia.org

Page 6 Page 3

PC PROJECT CHARITY GOLF TOURNAMENT 2011

WELCOME

We welcome you to the first Pachyonychia Congenita Charity Golf Tournament. We

hope you will make this an annual event—the funds raised will make a real difference in

advancing the most advanced in research in keratin disorders in the world.

Pachyonychia Congenita Project was founded in Salt Lake City in November 2003 and

the First International Symposium on Pachyonychia Congenita, headed by Sancy Leach-

man, MD, PhD, was held in February 2004 in Park City. At that meeting, the Interna-

tional PC Consortium (IPCC) was formed which has now grown to include over 100

physicians and scientists from more than 30 countries. In 2008, the first Phase 1b Clini-

cal Trial for PC was successfully completed proving the efficacy of a new class of drugs

known as siRNA (see abstract page 6 in this program).

From a base of only three patients in 2004, the PC Project registry now includes over

1000 patients in over 50 countries; over half (520) have now completed genetic testing.

This patient base and the collaboration of physicians and scientists has led to several

major 2011-2012 goals including a goal to sponsor multi-center, international clinical

studies for PC. Even though many of the specialists donate their time for our cause, the-

se trials cost millions of dollars.

Fundraising is key to accomplishing all that is now in envisioned. We appreciate so

much the sponsors of this 2011 Charity Golf Tournament and your individual participa-

tion. The funds raised (and the $2-for-$1 matching challenge) will help continue this

unique approach to patient advocacy and collaboration in research. We know our suc-

cesses will benefit many beyond the Pachyonychia Congenita community.

Holly Evans, Administrative Assistant

Mary Schwartz, Director

The phenotypic and molecular genetic

features of pachyonychia congenita.

McLean WH, Hansen CD, Eliason MJ, Smith FJ. Abstract Pachyonychia congenita (PC) is an autosomal dominant genodermatosis caused by heterozy-gous mutations in any one of the genes encoding the differentiation-specific keratins K6a, K6b, K16, or K17. The main clinical features of the condition include painful and highly debilitating plantar keratoderma, hypertrophic nail dystrophy, oral leukokeratosis, and a variety of epider-mal cysts. Although the condition has previously been subdivided into PC-1 and PC-2 sub-types, the phenotypic characterization of 1,000 mutation-verified PC patients enrolled in the International PC Research Registry, coordinated by the patient advocacy group PC Project, shows that there is considerable overlap between these subtypes. Thus, a new genotypic no-menclature is proposed, in which PC-6a represents a patient carrying a mutation in the K6a gene, etc. Although a rare disorder, PC represents a good model for therapy development, and international efforts are ongoing to develop and deliver siRNA, gene, correction, small mole-cule, and other strategies to treat this painful, disabling skin condition. The special relationship between PC Project and the PC research community has greatly accelerated the development pathway from gene identification to clinical trials in only a few years and represents a paradigm of hope for other orphan diseases.

J Invest Dermatol. 2011 May;131(5):1015-7. 2011 Mar 24.

PC PROJECT CHARITY GOLF TOURNAMENT 2011

PC PROJECT CHARITY GOLF TOURNAMENT 2011

First-in-human mutation-targeted siRNA phase

Ib trial of an inherited skin disorder.

Leachman SA, Hickerson RP, Schwartz ME, Bullough EE, Hutcherson SL, Boucher KM, Hansen CD, Eliason MJ, Srivatsa GS, Korn-brust DJ, Smith FJ, McLean WI, Milstone LM, Kaspar RL. Abstract The rare skin disorder pachyonychia congenita (PC) is an autosomal dominant syndrome that includes a disabling plantar keratoderma for which no satisfac-tory treatment is currently available. We have com-pleted a phase Ib clinical trial for treatment of PC utilizing the first short-interfering RNA (siRNA)-based therapeutic for skin. This siRNA, called TD101, specifically and potently targets the keratin 6a (K6a) N171K mutant mRNA without affecting wild-type K6a mRNA. The safety and efficacy of TD101 was tested in a single-patient 17-week, pro-spective, double-blind, split-body, vehicle-controlled, dose-escalation trial. Randomly assigned solutions of TD101 or vehicle control were injected in sym-metric plantar calluses on opposite feet. No adverse events occurred during the trial or in the 3-month washout period. Subjective patient assessment and physician clinical efficacy measures revealed re-gression of callus on the siRNA-treated, but not on the vehicle-treated foot. This trial represents the first time that siRNA has been used in a clinical setting to target a mutant gene or a genetic disorder, and the first use of siRNA in human skin. The callus re-gression seen on the patient's siRNA-treated foot appears sufficiently promising to warrant additional studies of siRNA in this and other dominant-negative skin diseases. Mol Ther. 2010 Feb;18(2):442-6. 2009 Nov 24.