Cure SMA, a non-profit organization dedicated to funding groundbreaking research on treatments for SMA, and providing families affected by this genetic disease to support they needs, is excited to welcome Dr. Mary Schroth, a medical professional education consultant, to their team. She will be working with Cure SMA a few days a month, and will retain her position as chair of the Medical Advisory Council.
Dr. Schroth will supervise the company’s patient care area, which dispenses valuable information on the disease through the care series booklets. While her expertise in patient care is very valuable to Cure SMA, having her on the team is especially beneficial as she takes the time to advise and educate other healthcare professionals on the disease.
While Cure SMA still devotes much of its efforts in facilitating groundbreaking research for a cure, the focus is still mostly on treatments that can inhibit disease progression, helping patients live a fuller, longer life. Those interested in connecting their healthcare providers with Cure SMA’s educational programs, can send an email to [email protected].
SMA (spinal muscular atrophy) is a genetic disease that affects the motor nerve cells in the spinal cord, gradually reducing one’s ability to walk, eat, and eventually, breathe. Today, it ranks as the leading genetic cause of death for infants.
In a previous report on SMA, Isis Pharmaceuticals, a Ribonucleic acid-targeted (RNA-targeted) drug discovery and development company, recently updated the results from an ongoing open-label Phase 2 trial of ISIS-SMNRx in infants with a diagnosis of spinal muscular atrophy (SMA) type I.
The company had previously reported results from this trial which included assessments of muscle function, event-free survival and evaluations of developmental milestones.
Results from ISIS-SMNRx tolerability and safety profile further support for the drug development. The company is working alongside Biogen to develop and commercialize ISIS-SMNRx as a treatment option for SMA patients.