Adopting some of the strategies behind successfully treating the childhood disease spinal muscular atrophy may enable development of therapies to curb the muscle decline that accompanies aging, new research suggests.
Nov 15, 2022
0
13
Spinal muscular atrophy (SMA), the most common form of neurodegenerative disease in childhood, is caused by defects in the SMN1 gene. SMA patients potentially benefit from three recently approved disease-modifying therapies. ...
Nov 7, 2022
0
14
The lone volunteer in a unique study involving a gene-editing technique has died, and those behind the trial are now trying to figure out what killed him.
Nov 4, 2022
0
60
There are numerous severe, difficult-to-treat monogenetic diseases that are caused by a defect in a single gene—also in the liver. These include the blood coagulation disorders hemophilia A or B or the metabolic disease ...
Oct 26, 2022
0
12
Muscular strength and its improvement are important to everyone for their regular day-to-day life activities. Conventionally, muscle function can be assessed by means of physical performance tests and/or muscle strength measurements. ...
Oct 12, 2022
0
3
Duchenne muscular dystrophy (DMD) is caused by a genetic mutation and affects one in every 5,000 boys born. Because the affected gene is on the X chromosome, girls are carriers of the mutant gene but develop the disease only ...
Sep 27, 2022
0
982
Mutations can be good and bad. Sometimes they help an organism adapt and survive. Other times they are so harmful that an organism can't survive or reproduce. Cold Spring Harbor Laboratory (CSHL) Professor Adam Siepel's team ...
Sep 22, 2022
0
44
Researchers at Johns Hopkins Medicine report that an experimental drug first developed to treat kidney disease prolongs survival and improves muscle function in mice genetically engineered to develop a severe form of Duchenne ...
Sep 13, 2022
0
14
For boys with Duchenne muscular dystrophy (DMD), vamorolone, a structurally unique dissociative steroidal anti-inflammatory drug, is effective and safe over a 24-week period, according to a study published online Aug. 29 ...
Sep 1, 2022
0
3
Many people have experienced the discomfort of food or a beverage accidentally going to the wrong place when swallowing. But swallowing issues sometimes become chronic and may be a sign of a health condition that should be ...
Aug 24, 2022
0
36
