COALITION TO CURE CALPAIN 3's Fundraiser:

C3 Adopt a Research Project

COALITION TO CURE CALPAIN 3's Photo
COALITION TO CURE CALPAIN 3's Photo
COALITION TO CURE CALPAIN 3's Photo
COALITION TO CURE CALPAIN 3's Photo
COALITION TO CURE CALPAIN 3's Photo
COALITION TO CURE CALPAIN 3's Photo

COALITION TO CURE CALPAIN 3

Dr. Melissa Spencer @ UCLA, Dr. Lou Kunkel @ Boston Children's & Dr. Isabelle Richard @ Genethon have begun research projects funded by C3!

via Crowdrise 12 months ago

THE STORY:

Limb-girdle Muscular Dystrophy type 2A  (LGMD2A), also known as calpain 3 deficiency or calpainopathy, is a progressive disease that causes muscle weakness so severe that those who have it are eventually confined to a wheelchair. But because it is a rare disease and -- although debilitating -- is not life-threatening, it attracts significantly fewer research dollars and thus fewer researchers working to understand the disease and discover a cure. Yet, from the limited amount that is known about calpain 3 deficiency, according to leading researcher Dr. Eric Hoffman, “…it seems to be one of the most 'curable' of the muscular dystrophies”.

Glaring Unmet Needs:
LGMD2A is not completely understood and there is no cure or even a treatment; there had never been a workshop held specifically for LGMD2A researchers; there was no nationally recognized patient registry and therefore, no list of people for researchers to contact about clinical trials of promising therapies; there is little awareness of LGMD2A among the general public.

Coalition to Cure Calpain 3 was founded in October of 2010 to address these unmet needs.  Since that time, our volunteer organization has:

  • attracted a Scientific Advisory Board that includes Drs. Kevin Campbell, Eric Hoffman and Melissa Spencer
  • organized the first ever U.S. workshop focused solely on LGMD2A/Calpainopathy held on October 27, 2011 in Santa Monica, California and followed up with an international workshop co-sponsored with the European Neuromuscular Centre in Amsterdam in November 2013
  • in partnership with Melissa Spencer, applied for and been awarded an MDA grant to help fund the conference
  • created the first nationally recognized patient registry for LGMD2A
  • begun to build awareness of LGMD2A and unite the community by designing and launching our website (www.curecalpain3.org) and by becoming active on the social networking sites of Facebook and Twitter

Adopt a Research Project:
Based on the outcomes of our October scientific workshop where international experts in calpain 3 research shared knowledge and strategized future directions for basic research and therapeutic/diagnostic development, the C3 Scientific Advisory Board and Board of Directors have awarded three grants to support the most promising research projects: Dr. Melissa Spencer (UCLA): "Exploring the Role of Blunted Signaling Pathways in the Pathogenesis of Calpainopathy"; Dr. Lou Kunkel (Boston Children's Hospital): "Using Transcription Activator-like Nucleases (TALENs) to Create a Calpain-3 Null Zebrafish for Subsequent Characterization and Screening for Small Molecule Corrective Agents; and Dr. Isabelle Richard (Genethon): "AAV-mediated Transfer of Calpain 3".

These projects were funded by donations made by those of us who are touched by LGMD2A/calpainopathy in some way as well as those who are not associated with this disease in any particular way but who are moved to support efforts to cure it.

Your support is critical as we continue to focus on research efforts to cure this devastating disease.

We thank you for considering to adopt part of this project as your own and asking your friends and family to do the same. As a community standing together, we have the power to find a cure or a treatment for this devastating disease.

With much gratitude,

Michele Wrubel, Co-founder

DONATE

To This Fundraiser

$39,998

MONEY RAISED
  •  
  •  
  •  
  •  
  •  
  •  
  •  
  •  
  •  
  • The Birk Family

    $100

  • Mark and Elizabeth

    $200

  • The Navarro family

    $100

  • the greenspun family

    $50

  • Lisa Metz

    $50

  • Darcy Hicks

    $300

  • Joe and Maureen

    $300

  • Donna and David Hyman

    $100

  • Barbara & Alan Phillips

    $100

  • Susan Terry

    $100

  • Naomi and Jeff Weingart

    $200

  • Jody and Joel Beck

    $100

  • DeSouza-...

    $100

  • Cari and Keith Kaplan

    $100

  • Linda and Len Russ

    $100

  • Moran Family

     

  • Landau Family

    $180

  • Daniel and Diana Sussman

    $100

  • The Kleinmans

    $540

  • The Haroun Family

    $500

  • The Radin Family

    $100

  • Ellen and Art Gang and children

    $150

  • Kenny and Wendy Epstein

    $100

  • Anne and Todd Cheema

    $250

  • The Mirkine Family

    $100

  • Suzanne & Peter Propp

     

  • Rosen Family

    $100

  • Cooperst... Family

    $100

  • The Honigstein Family

     

  • The Folger Family

     

  • Craig Pastolove

    $250

  • The Book Family

    $100

  • Ann Breslauer

    $100

  • Robin and Brian Kurtz

    $100

  • The Popkin Family

    $100

  • Susan Klimist

    $50

  • Jane Tewksbury

    $125

  • Bob and Cheri Tewksbury

    $1,000

  • Debra & Edward Smolka

    $100

  • Frances Rowland

    $500

  • Pamela and David Furth

    $180

  • VALERIE SEILING JACOBS

    $100

  • Shmuel & Rivka Bernstein

    $180

  • Laura and Lanning Bryer

    $100

  • Daffan Family

    $100

  • Margie and Bob Labarre

    $180

  • Anita & Ari Rothman

     

  • Narendra Gupta

    $5,000

  • Judy and Steve Samuels

    $250

  • The Frost Family

    $136

  • Faith Taylor and Ted Sullivan

    $50

  • Benay & Steve Rubin

     

  • Becky and Mike Goss

     

  • Candi Savin & Dan Gross

    $150

  • martina and bob israely

    $50

  • Gwen & David Baker

    $600

  • Nick & Karin Curci

    $100

  • Sharon and Jim

    $100

  • Nancy & Spencer Hart

    $500

  • Lisa and Adam Hodes

    $500

  • Rod Sweeny

    $50

  • The Rozynek Family

    $100

  • Anonymous

    $500

  • Anne & David Tetenbaum

    $50

  • Beverly Tewksbury

    $500

  • Robert Tewksbury

    $500

  • Gwen and Leanne

    $500

  • Ronna and Marc Zaken

    $100

  • Karin Fraade

    $100

  • The Paukers

     

  • Laura & Mitch Felton

    $180

  • Austin Wrubel

    $125

  • A Friend of C3

    $5,000

  • Gwen & David Baker

    $600

  • Evelyn Wrubel

    $3,600

  • Lisa & Ed Raice

    $500

  • Nancy & David Berkowitz

     

  • Nancy and Josh Cohen

    $136

  • Hilary & Roger Machlis

     

  • The Liebergall Family

    $250

  • Shelly & Michael Kassen

    $1,800

  • Mona Kurnit

     

  • Wendy and Jeff Cohen

    $100

  • Anonymous

     

  • Anonymous

    $50

  • Jennifer & Alan Clement

    $250

  • Marianne and Tim Goodell

    $100

  • Johannes Hoeggerl

    $150

  • The Greenspan Family

     

  • Risa Glick

     

  • Gilda Glick

     

  • Pablo Soto

    $500

  • Stéphane M. Grueso

    $50

  • Irene

    $100

  • Stamatis Kiriazis

    $100

  • Morgaine & Mark Pauker

    $100

  • Jack Harryman

    $200

  • Paige and Griffin Cogorno

    $200

  • Anonymous

    $500

  • Anonymous

    $100

  •  
  •  
  •  
 

80% Raised of $50,000 Goal

Fundraise for this Campaign

The Team: $39,998 TOTAL RAISED SO FAR

JOIN THE TEAM
Fundraiser Title

COALITION TO CURE CALPAIN 3

Amount Raised:

$38,748

 

77% Raised of $50,000 Goal

Fundraiser Title

Frances Rowland

Amount Raised:

$500

Fundraiser Title

leanne bell

Amount Raised:

$500

Fundraiser Title

michael gk

Amount Raised:

$150

Fundraiser Title

Alan Radin

Amount Raised:

$100

Fundraiser Title

Aurea De Souza

Amount Raised:

$0

Fundraiser Title

Linda Russ

Amount Raised:

$0

Fundraiser Title

Matthew Landau

Amount Raised:

$0

Donor Comments

Anonymous

ANONYMOUS

DONATION: $10

2 years ago

Lisa Bowman

Lisa Bowman

DONATION: $2,500

In honor and celebration of my dear friend, Michele. 3 years ago

The Wright Family

The Wright Family

DONATION: $100

3 years ago

Anonymous

ANONYMOUS

DONATION: $10

3 years ago

Anonymous

ANONYMOUS

DONATION: $50

3 years ago