by Nisha Cooch, PhD | Sep 10, 2019 | Neurologists Resource Page One, Neurologists Resource Pages, Nursing Resource Page One, Nursing Resource Pages, Pediatricians Resource Page One, Pediatricians Resources, Radiology Resource Page One, Radiology Resources
Zolgensma, or onasemnogene abeparvovec, is an adeno-associated virus vector-based gene therapy that was approved by the FDA in May 2019. Given the newness of this therapy, it is important that healthcare providers are educated on specific information regarding the...
by Nisha Cooch, PhD | Jan 28, 2019 | Genetic Counselor Resource Page One, Genetic Counselor Resource Pages, Neurologists Resource Page One, Neurologists Resource Pages, Nursing Resource Page One, Nursing Resource Pages, Nutritionist Resource Page One, Nutritionist Resource Pages, Orthopedics Resource Page One, Orthopedics Resource Pages, Pediatricians Resource Page One, Pediatricians Resources, Physical Therapists Resource Page One, Physical Therapists Resource Pages, Pulmonologist Resource Page One, Pulmonologist Resource Pages, Radiology Resource Page One, Radiology Resources, Social Worker Resource Page One, Social Worker Resource Pages, Urologist Resource Page One, Urologist Resources
At the end of 2016, the U.S. Food and Drug Administration (FDA) approved nusinersen for the treatment of spinal muscular atrophy (SMA).1 Because of the high threshold for FDA approval, an abundance of data on nusinersen and its physiological effects on SMA patients...
by Michael Nace | Jan 15, 2019 | Neurologists Resource Page One, Neurologists Resource Pages
In 2016, nusinersen, or Spinraza, became the first drug for spinal muscular atrophy (SMA) ever approved by the U.S. Food and Drug Administration.1 The drug acts to combat low survival motor neuron (SMN) protein in the central nervous system by correcting the splicing...
by Michael Nace | Jan 7, 2019 | Genetic Counselor Resource Page One, Genetic Counselor Resource Pages, Neurologists Resource Page One, Neurologists Resource Pages
Mutations in the survival motor neuron 1 gene (SMN1), which result in reduced expression and lower levels of the full-length SMN protein, are responsible for spinal muscular atrophy (SMA).1 Given the role that microRNAs (miRNAs) play in gene expression, it is perhaps...
by Michael Nace | Dec 19, 2018 | Genetic Counselor Resource Page One, Genetic Counselor Resource Pages, Neurologists Resource Page One, Neurologists Resource Pages
The therapies that are being developed for spinal muscular atrophy (SMA) generally fall into two categories: those that aim to increase levels of survival motor neuron (SMN) protein and those that are SMN-independent.1 The therapies that aim to increase SMN protein...
by Michael Nace | Nov 27, 2018 | Neurologists Resource Page One, Neurologists Resource Pages, Nursing Resource Page One, Nursing Resource Pages, Orthopedics Resource Page One, Orthopedics Resource Pages, Pediatricians Resource Page One, Pediatricians Resources, Physical Therapists Resource Page One, Physical Therapists Resource Pages
Increased pain is significantly associated with lower levels of health, social function, and vitality.1 The results of studies on animal models of neuropathic pain suggest that the abnormal excitability of dorsal horn neurons in spinal muscular atrophy (SMA) may lead...
