FDA-approved treatments for spinal muscular atrophy differ in their mechanisms of action, safety profiles, and administration ...

The company confirmed today that regulators in the US and Europe have started reviews of a new regimen based on two 50mg doses of Spinraza (nusinersen) given 14 days apart, followed by a higher ...

At the moment, the only other approved medicine for SMA is Biogen’s blockbuster SMA treatment Spinraza (nusinersen), so it will be interesting to see if Zolgensma’s availability has impacted ...

Most adolescents and adults with SMA have normal sensory nerve function, even those with long-lasting disease, a study finds.

Its products include TECFIDERA, VUMERITY, AVONEX, PLEGRIDY, TYSABRI and FAMPYRA for the treatment of MS, SPINRAZA for the treatment of SMA, ADUHELM for the treatment of Alzheimer's disease ...

Cancer continues to be one of the leading causes of death worldwide, and conventional cancer therapies such as chemotherapy, radiation therapy, and surgery have limitations. RNA therapy and cancer ...