조직 이름: Coalition to Cure Calpain 3
웹 사이트: CureCalpain3.org
페이스북: Coalition to Cure Calpain 3: Overcoming LGMD2A/Calpainopathy
Twitter: @CureCalpain3
WHAT LGMD SUB-TYPE DOES YOUR ORGANIZATION FOCUS ON:
LGMD2A, a form of Calpainopathy
하위 유형에 초점을 맞추는 경우 레지스트리가 있으며 환자는 어떻게 가입할 수 있나요?
Yes, we maintain the global LGMD2A Patient Registry. It’s easy to sign up at www.lgmd2a.org
귀사는 비영리 단체인가요? 그렇다면 어떤 유형인가요?
Yes, we are a U.S.-based 501 (c)(3) tax-exempt public charity
조직을 만들게 된 계기:
Coalition to Cure Calpain 3 (C3) was founded in 2010 for the specific purpose of funding research efforts focused on understanding the biology of and finding a cure for LGMD2A, a form of Calpainopathy. This organization was created by patients and their families who were frustrated with the lack of knowledge about the disease and the fact that very little research was being done. For some of us, even getting a specific diagnosis had taken decades. We are motivated by our desire to encourage collaboration among scientists, those who have LGMD2A, their families and friends, and the community-at-large to bring an end to this under-researched, underfunded “orphan” disease.
조직의 미션은 무엇인가요?
C3 is committed to treating and ultimately curing limb girdle muscular dystrophy type 2A (LGMD2A), a form of Calpainopathy. Our mission is to fund high potential research and clinical trials as we educate the global community about this disease.
조직이 제공하는 서비스
– Fund research focused on LGMD2A/Calpainopathy
– Maintain the global patient registry for LGMD2A/Calpainopathy
– Educate the global community (including the scientific community along with patients, their families, and the general public) about the disease via our website, social media, presence at scientific conferences, and email communications
– Provide a support forum for patients and their families on Facebook (email C3 Director of Patient Outreach Carol Abraham to join)
귀사의 가장 자랑스러운 점은 무엇인가요?
We are most proud of the fact that we have been able to attract and support high caliber scientists to spur calpainopathy research. Dr. Melissa Spencer has led our Scientific Advisory Board since our inception in 2010, working alongside Drs. Kevin Campbell, Eric Hoffman, and Lou Kunkel. Dr. Jennifer Levy joined us as the first C3 Scientific Director in 2016 and has more than doubled the number of research projects we are funding. The fact that our foundation, initially just an idea we had to try to make a difference, has committed almost $1.5 million dollars to calpainopathy research undertaken by leading scientists – Drs. Michele Calos, Lou Kunkel, Cathleen Lutz, Rita Perlingeiro, Isabelle Richard, Melissa Spencer, Volker Straub – is something we are indeed proud of.
귀사에 대해 전 세계가 알았으면 하는 것은 무엇인가요?:
We recently launched a Gene Therapy Initiative that seeks to accelerate the understanding of gene therapy as a treatment for LGMD2A by funding the research of multiple gene therapy approaches. If results from any current or future Gene Therapy Initiative research projects are positive, they can be progressed towards the initiation of clinical trials for LGMD2A patients. Be on the lookout for announcements from us as we continue to fund new projects. http://www.curecalpain3.org/research
사람들이 조직을 지원하는 데 어떻게 참여할 수 있을까요?
Bringing a therapy from the research lab to a clinical trial is a long and expensive process. The most critical way people can support our organization’s mission is to donate. Every dollar makes a difference in our efforts to cure this muscle-wasting disease.
Donors can make donations in three ways:
– our website (online)
– our Crowdrise campaign (online)
– by mail via check (offline)
email us and ask for a donation form or download one at http://files.constantcontact.com/fc88c999301/4a707677-bb13-4ccf-a5a7-d75c7630cb0e.pdf
조직에 연락하는 가장 좋은 방법은 무엇인가요?
Email us 에서 info@curecalpain3.org. We will reply!
추가하고 싶은 내용이 있나요?
If you know that you have some form of limb girdle muscular dystrophy, please push for genetic testing to identify your type. There is still much for all of the LGMD organizations and the scientific community to learn about this group of diseases, and you can help by joining the appropriate patient registry. Seek out the many support groups; you are not alone. Please know that we are motivated and working every day to learn more about calpainopathy and to find a treatment or cure.
