Preliminary Study Suggests Drug May Help Babies with Spinal Muscular Atrophy
A preliminary study suggests that an investigational drug may help increase protein levels in babies with spinal muscular atrophy.
Spinal muscular atrophy (SMA) is an inherited disease that leads to loss of motor function. It is the leading genetic cause of death in infants and toddlers. The disease is caused by reduced levels of the survival motor neuron (SMN) protein. In SMA, the SMN1 gene is mutated or missing. The backup SMN2 gene allows production of some of the necessary protein.
The new drug, called RG7916, is a liquid solution given orally once a day. It is designed to modulate the SMN2 gene splicing to increase SMN protein.
Click here to read more.
2018 Advanced Endoscopic Skull Base and Pituitary Surgery
June 1-2, 2018; New York
2018 American Society for Stereotactic and Functional Neurosurgery Biennial Meeting
June 2-5, 2018; Denver
Complex Endoscopic Endonasal Surgery of the Skull Base
June 7-9, 2018; Pittsburgh, PA
2018 Annual Meeting of the Michigan Association of Neurological Surgeons
June 8-10, 2018; Thompsonville, MI
CARS 2018 Computer Assisted Radiology and Surgery
June 20-23, 2018; Berlin, Germany
Be the first to reply using the above form.