Web Results
patienteducation.asgct.org/disease-treatments/sma

Sep 13, 2021 ... Gene therapy is a fitting approach for diseases caused by a single gene mutation, like SMA. It targets the cause of disease by delivering a ...

www.cuimc.columbia.edu/news/spinal-muscular-atrophy-new-clues-cause-and-treatment

May 15, 2017 ... SMA is triggered by mutations in a gene called SMN1 (Survival Motor Neuron 1), causing a deficiency of SMN protein in all cells—including spinal ...

smanewstoday.com/social-clips/2017/04/11/explaining-causes-symptoms-treatment-sma

Apr 11, 2017 ... This animated video illustrates the neurons involved in the genetic disease spinal muscular atrophy (SMA) and how they affect the muscles of ...

journals.biologists.com/dmm/article/10/8/943/53035/Therapeutic-strategies-for-spinal-muscular-atrophy

Aug 1, 2017 ... Spinal muscular atrophy (SMA) is a devastating neuromuscular disorder characterized by loss of motor neurons and muscle atrophy, ...

pediatricmotordisorders.org/disorders/sma

Symptomatic individuals of all ages can be tested through DNA studies typically done from a blood sample. SymptomsDiagnosisManagement & TreatmentAdditional ...

www.medicinenet.com/spinal_muscular_atrophy_sma_symptoms_and_signs/symptoms.htm

Sep 17, 2019 ... Learn about spinal muscular atrophy (SMA) symptoms and signs and the medications that treat it. Symptoms and signs include breathing ...