Alliance Against HMERF Muscular Dystrophy reflects the growth of the Foundation for Casey's Cure.
With an expanded goal of connecting the entire HMERF population, we ask you to join us in our mission to accelerate groundbreaking research, drive innovation, and create transformative treatments for HMERF muscular dystrophy.
While many researchers are studying the mechanisms of the HMERF gene, we are the only organization in the world actively seeking a life saving treatment to ensure that no one faces HMERF without hope. Many of these leading researchers are on our Scientific Advisory Board.
By uniting scientists and healthcare professionals around the world, we are accelerating the advancement of pioneering medical breakthroughs that will lead to therapeutic treatments that will help thousands of lives.
Make a difference in the lives of those battling a rare disease.
Join us in providing hope and support for those who need it most
A life-saving answer to HMERF is within our reach, and we are making great strides. Because of you, we can make a difference and continue our relentless pursuit of developing a therapeutic for HMERF.
HMERF is a rare genetic muscle disorder that primarily affects skeletal muscles and respiratory function. It is caused by mutations in the TTN (titin) gene which plays a crucial role in muscle structure and elasticity.
This type of muscular dystrophy causes a progressive loss of muscle mass, eventually leaving patients to become bedridden and in severe pain. Ultimately, the disease will determine how long each patient can continue to breathe on their own. For each patient, and for unknown reasons, the path through this disease can be very different.
Many people confuse muscular dystrophy (MD) with multiple sclerosis (MS). A simple way to remember the difference is that MD stands for “Muscle Death.” Once muscles die, they cannot regenerate—at least not yet—which makes every moment crucial.
People with HMERF live their lives on a ticking clock, but with your help and support, we can provide patient support and push the science forward to fight back against this devastating disease.
Casey’s story is much like other people with HMERF. A disease that will take her life because the muscles in her body are dying.
A nurse in an outpatient clinic at WellStar Pulmonary Medicine in Georgia, USA, Casey married her high school sweetheart, Josh, and together they have two sons, Joshua and Ethan.
In 2016, Casey was diagnosed with HMERF, and their lives changed forever.
We are proud to bring together leading medical science experts from around the world to lead our efforts in developing a therapeutic cure for HMERF.
Dr. Savarese is the top HMERF geneticist who works with the leading global HMERF researcher, Dr. Udd, overseeing three ongoing studies in Finland, France, and Israel.
Dr. Lindquist research focuses on congenital myopathies such as nemaline myopathies and titinopathies such as HMERF.
Dr Pfeffer’s research focuses on creating a methodology to repair the damaged gene.
Dr Cagnin is an experienced PHD skilled in Sequence Analysis, DNA Sequencing, Bioinformatics, Real-Time Polymerase Chain Reaction (qPCR), and Systems Biology.
Dr Sales is a computational biologist interested in developing highly efficient methods for analyzing large datasets generated by high‑throughput experimental techniques.
Dr Novak brings strong expertise in developing specific therapeutics for other types of muscular dystrophy and is providing invaluable advice as we move toward developing a therapeutic for Casey and other HMERF patients.
Read our progress updates as we get closer to fullfilling our mission.
