The Food and Drug Administration on Friday approved the first treatment for children and adults with spinal muscular atrophy, a rare genetic disorder marked by progressive muscle weakness that's the most common genetic cause ...
Dec 23, 2016
0
0
The commonly-used epilepsy drug, valproic acid (VPA), can have a highly beneficial effect on some babies born with spinal muscular atrophy (SMA), the number one genetic killer during early infancy. But in about two-thirds ...
Jun 23, 2012
0
0
A preliminary study suggests that an investigational drug may help increase protein levels in babies with spinal muscular atrophy. The open-label study is released today and will be presented at the American Academy of Neurology's ...
Apr 18, 2018
0
4
The lab of Yongchao C. Ma, Ph.D., at Stanley Manne Children's Research Institute at Ann & Robert H. Lurie Children's Hospital of Chicago has discovered a fundamental biological mechanism that could lead to new treatments ...
Apr 5, 2024
0
11
This week, scientists gathered in Washington, DC for the International Summit on Human Gene Editing to discuss a technology called CRISPR-CAS9, which can insert, remove and change the DNA of basically any organism. It is ...
Dec 4, 2015
0
21
Today, a team of researchers from Cold Spring Harbor Laboratory (CSHL) sheds new light on the underlying pathology of spinal muscular atrophy (SMA), a rare but devastating disease that causes muscle weakness and paralysis ...
Jan 12, 2015
1
35
The Editor-in-Chief of Human Gene Therapy, the first journal devoted to the field of gene therapy, and one of the world's leading experts on gene therapy have co-authored a new editorial, Moving Forward After Two Deaths in ...
Jul 2, 2020
0
0
(Medical Xpress)—Reviving a gene which is 'turned down' after birth could be the key to treating Duchenne muscular dystrophy (DMD), an incurable muscle-wasting condition that affects one in every 3,500 boys.
Dec 2, 2013
0
0
(Medical Xpress)—For decades, scientists have searched for treatments for myopathies – genetic muscular diseases such as muscular dystrophy and ALS, also called Lou Gehrig's disease. Now, an interdisciplinary team of ...
Nov 4, 2013
0
0
Using fruit flies, Texas A&M AgriLife Research scientists may be one step closer to better understanding the pathological mechanisms of muscular dystrophy.
Mar 28, 2018
0
57
