Sharevault Supports Cure SMA and Its Dedication To Curing Spinal Muscular Atrophy03 April, 2017
SMA (spinal muscular atrophy) is a disease that robs people of physical strength by affecting the motor nerve cells in the spinal cord, taking away the ability to walk, eat or breathe. It is the number one genetic cause of death for infants.
SMA is caused by a mutation in the survival motor neuron gene 1 (SMN1). In a healthy person, this gene produces a protein that is critical to the function of the nerves that control our muscles. Without it, those nerve cells cannot properly function and eventually die, leading to debilitating and often fatal muscle weakness.
SMA affects approximately 1 in 10,000 babies, and about 1 in every 50 Americans is a genetic carrier.
Cure SMA (curesma.org) is a patient advocacy group dedicated to the treatment and cure of SMA by funding groundbreaking research that drives breakthroughs in treatment and care and providing support to affected families. There are two sides to the organization. The first is the patient support side. Cure SMA currently has over 12,000 households in their membership database and 115,000 donors. There are also over 35 local chapters nationwide. Cure SMA supports families by providing them with information on SMA when they first get a diagnosis and information about how to care for the family member who has the disease. They provide them with ongoing clinical trial information and other research that is in progress. They also give them a packet of useful toys and household items that are tailored to their child’s functional level as well as providing some medical equipment.
The other side of the organization is the research side, where they fund and support research that is exploring a cure for SMA. Cure SMA has committed over 62 million dollars to research since the organization began.
ShareVault is a proud sponsor of Cure SMA, providing a free data room that Cure SMA uses to license proprietary technologies they own.
Cure SMA has their own series of molecules that they outlicense to potential partners to further treatment for SMA.
In December of 2016, the U.S. Food and Drug Administration (FDA) approved Biogen’s SPINRAZA™ (nusinersen) under Priority Review for the treatment of SMA in pediatric and adult patients. SPINRAZA is the first and only treatment approved in the U.S. for SMA.
Cure SMA also hosts an annual conference, which brings together researchers, healthcare professionals and families to network, learn and collaborate. The family conference includes a variety of workshops, keynote sessions with leading researchers, a family-friendly research poster session, and more—plus fun events like a dance party, a meet-and-greet, a pajama party and movie night, and teen and adult social activities.
The Annual SMA Conference is really two separate events—one for families and one for researchers—that run in parallel, so that families, researchers, and clinicians can interact with and learn from one another.