Updated: Feb 18
The 11-Year-Old Nonprofit Will Receive a Two-Year Grant for Capacity-Building Efforts
LAKEWOOD, CA, USA (Feb. 3, 2020) — Cure CMD is thrilled to announce that it has been selected as one of 30 grantees as part of the Chan Zuckerberg Initiative’s (CZI) Rare As One Project, aimed at supporting and lifting up the work that rare disease-affected communities are doing to accelerate research and drive progress. In total, the Chan Zuckerberg Initiative has awarded $13.5 million in funding to patient-led organizations that are working to find treatments and cures for rare disease.
Rare disease is anything but rare: as many as 7,000 rare diseases affect 400 million people worldwide. The vast majority of these diseases are not well known or understood, and fewer than five percent have any FDA-approved therapy. The knowledge and learnings of affected individuals are key to driving breakthroughs in research and treatment, but a lack of funding and infrastructure to support such patient-led research is holding that progress back.
“We are honored to be a part of the communities that make up this innovative project, critical to advancing research in rare disease and specifically for us, in Congenital Muscular Dystrophy,” says Rachel Alvarez, Executive Director of Cure CMD. “For more than a decade we’ve been making a concerted effort to move the needle, and this grant will be an important step in our progress.”
CZI will provide Cure CMD, along with 29 other patient organizations, with funding, training, community mentorship, and capacity-building services to assist in establishing a network of researchers and clinicians, convening their community, and further developing a research agenda. The goal is that these organizations will share feedback with and learn from one another, and identify how to best address the most pressing needs across a spectrum of rare diseases. View the full list of grantees.
Cure CMD Scientific Director, Dr. Gustavo Dziewczapolski, says, “The confluence of significant progress in research toward treatments, plus this investment to build Cure CMD’s capacity, makes for a promising next chapter for Congenital Muscular Dystrophy.”
Cure CMD’s community-focused vision is to apply CZI support to 1) create a lasting, momentum-building infrastructure for our research community that is directly informed by the needs of affected individuals; 2) launch retrospective pulmonary and nutrition studies to inform near-future prospective studies; 3) initiate pulmonary traveling clinics to educate non-expert respiratory clinicians how to recognize and treat early signs of respiratory weakness in neuromuscular patients, and aid local institutions in developing transitions programs so that care remains as consistent as possible into adulthood; and 4) hold a scientific and family conference toward these efforts. Long-term goals include new research projects and lasting organizational sustainability.
“No one is more committed to finding cures for rare diseases than the patients and families of those affected by these disorders,” said Priscilla Chan, Co-Founder & Co-CEO of CZI. “We are proud to support patient-led organizations as they pursue diagnoses, information, and treatment options in partnership with researchers and clinicians.”
“We’re excited to welcome this group of grantees — led by patients and caregivers with diverse backgrounds, perspectives, and expertise — to work together and learn together,” said Tania Simoncelli, CZI Science Policy Director and Rare As One Project lead. “We hope the support, training, and mentorship provided through CZI’s program will be transformative for these organizations and serve as a model that can be catalytic for other groups battling rare diseases.”
Cure CMD was founded in 2008 to advance research toward treatments for the congenital muscular dystrophies, and is committed to improving the lives of those living with CMD through engagement and support of the community. To date, the organization has co-funded more than $3 million in research, launched the Congenital Muscle Disease International Registry and two clinical trials, and connected more than 4,000 stakeholders worldwide, through the registry, social media, educational webinars, and conferences. Visit curecmd.org for more information.
CHAN ZUCKERBERG INITIATIVE
Founded by Dr. Priscilla Chan and Mark Zuckerberg in 2015, the Chan Zuckerberg Initiative (CZI) is a new kind of philanthropy that’s leveraging technology to help solve some of the world’s toughest challenges — from eradicating disease, to improving education, to reforming the criminal justice system. Across three core Initiative focus areas of Science, Education, and Justice & Opportunity, we’re pairing engineering with grant-making, impact investing, and policy and advocacy work to help build an inclusive, just and healthy future for everyone. For more information, please visit chanzuckerberg.com.