Spinal Muscular Atrophy
How We Can Help You
Stanford Neuromuscular Program has extensive experience in diagnosing and treating spinal muscular atrophy (SMA). We are at the forefront of developing and validating new treatment approaches, such as Spinraza, the only treatment available for spinal muscular atrophy.
This disease-modifying drug halts the progression of spinal muscular atrophy and may help people regain some of the motor function they have lost. Our research team treated the first infant ever to receive Spinraza in 2013. After the FDA approved Spinraza in 2016, we treated the very first adult living with spinal muscular atrophy.
We offer specialized, comprehensive care for those living with spinal muscular atrophy at our multidisciplinary Neuromuscular Clinic Program.
WHAT WE OFFER FOR SPINAL MUSCULAR ATROPHY
- Experience and expertise as the first medical center to treat an adult patient living with SMA using a disease modifying drug (Spinraza) our team helped develop.
- Advanced treatment options and clinical trials, including access to the only treatment that stops the SMA progression, as well as access to other experimental therapies.
- Multi-specialty team approach that brings together neuromuscular specialists, genetic counselors, dietitians, pulmonologists, and physical, occupational, respiratory, and speech therapists in one clinic.
- Comprehensive support provided by our social worker, community liaison and a Muscular Dystrophy Association (MDA) representative to connect you with supportive, community and disability resources.
TREATMENT FOR SPINAL MUSCULAR ATROPHY (SMA)
Our Neuromuscular Program doctors are recognized experts in caring for all types of spinal muscular atrophy.
As a designated Muscular Dystrophy Association Care Center, we provide a team-based approach to care that brings together many specialists, including neurologists, geneticists, pulmonologists, and physical, occupational, speech and language pathologists, as well as respiratory, and other therapists.
We work together in real-time to create a comprehensive care plan. We work closely with our colleagues in Pediatric and Adult Neurology to bring you the care you need. This allows for a seamless transition, and continuity of care, when children become adults and transfer from the children’s hospital to Stanford. In fact, many of our team members work at both the children’s clinic and the adult clinic.
Features of our clinic include:
- Comprehensive care in one location: At most appointments, you will see your care team of specialists, so you and the team can communicate together, in real time.
- Holistic approach: We bring you treatments and access to a wide range of specialists, such as behavioral therapists, to address aspects of the condition that do not directly affect the muscles. Also, you and your loved ones will be connected to community and financial resources and services when you meet the social worker and the community liaison.
- Newest available therapies: We are involved in pioneering research for treatments and therapies for SMA.
- Investigative therapies: At Stanford, you have opportunities to get involved in experimental treatments through participating in clinical research trials.
- Family support: We understand that conditions like SMA affect your entire family. We offer an experienced social worker and a community liaison to help connect you with supportive community resources.
SMA Drug Therapy
Our doctors helped develop a disease-modifying drug, Spinraza, and treated the first adult ever, after FDA approval. The drug helps the surviving motor neuron (SMN2) gene make the SMN protein work more efficiently and improve the health of the nerves that control muscles.
Determining Eligibility for Spinraza Treatment
Our team determines if you will be eligible for the drug after baseline tests, including a genetic test, imaging, labs, and physical therapy assessment, and we work with your insurance company to obtain approval. Once your insurance authorization is complete, you will receive four injections in the first two months and injections every four months after.
CLINICAL TRIALS FOR SPINAL MUSCULAR ATROPHY
Clinical trials are research studies that evaluate a new medical approach, device, drug, or other treatment. As a Stanford Health Care patient, you may have access to the latest, advanced clinical trials.
Open trials refer to studies currently recruiting participants or that may recruit participants in the near future. Closed trials are not currently enrolling, but similar studies may open in the future.
To learn more about the clinical trials we offer, contact Shirley Paulose at 650-724-3792.