Web Results
www.ajmc.com/view/improved-treatment-options-for-sma-present-new-opportunities-challenges

Sep 5, 2020 ... There are now multiple therapeutic options available for spinal muscular atrophy (SMA), a disease once considered incurable; however there ...

smanewstoday.com/sma-treatment

Aug 11, 2020 ... Spinraza (nusinersen), by Biogen, was the first disease-modifying therapy for SMA that the FDA approved in December 2016 to treat SMA in both ...

kidshealth.org/en/parents/sma.html

What Are the Signs and Symptoms of SMA? ... The signs of SMA can vary. Some babies with SMA are "floppy" and don't learn to roll or sit at the expected age. An ....

www.chop.edu/treatments/gene-therapy-spinal-muscular-atrophy-sma

Gene replacement therapy for spinal muscular atrophy (SMA) is now offered as a treatment option for children who meet certain criteria.

www.uptodate.com/contents/spinal-muscular-atrophy

Aug 6, 2021 ... Disease severity in SMA generally correlates inversely with SMN2 copy ... as a substitute for medical advice, diagnosis, or treatment.

stanfordhealthcare.org/medical-conditions/brain-and-nerves/spinal-muscular-atrophy.html

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 ...

medlineplus.gov/spinalmuscularatrophy.html

They are based on how serious the disease is and when the symptoms start: Type l is also called Werdnig-Hoffman disease or infantile-onset SMA.

medlineplus.gov/genetics/condition/spinal-muscular-atrophy

Individuals with spinal muscular atrophy type II cannot stand or walk unaided. They often have involuntary trembling (tremors) in their fingers, a spine that ...

www.healthline.com/health/spinal-muscular-atrophy/sma-treatment-options

SMA therapies · nusinersen (Spinraza), which is approved to treat SMA in children and adults · onasemnogene abeparvovec-xioi (Zolgensma), which is approved to .....

rarediseases.org/rare-diseases/spinal-muscular-atrophy

The following resource from Cure SMA provides a description of ... SMA type 0 is the most severe form of the disease and is ...

www.childrenshospital.org/conditions-and-treatments/conditions/s/spinal-muscular-atrophy-sma/diagnosis-and-treatment

As yet, there is no complete cure for SMA. However, the discovery of the genetic cause of SMA has led to the development of several treatment options that ...