Health
Pool neck floats are a danger to babies, FDA warns
Neck floats marketed for babies to use in water can lead to serious injury or death, the U.S. Food and Drug Administration warned this week.
Jun 29, 2022
0
2
Page 8 - News tagged with muscular atrophy
Neuroscience
Electroceuticals for the treatment of diseases caused by loss of myelin
Electrical stimulation of nerve fibers could promote the growth of the myelin sheath, a protective layer around neurons that is crucial for transmitting nerve impulses. The finding, published in the journal Biofabrication, ...
Mar 21, 2022
0
20
Oncology & Cancer
Cutting off liver cancer's nutrient supply chain
The World Health Organization projects that starting in 2030, over a million people will die each year from liver cancer. Cold Spring Harbor Laboratory (CSHL) Professor Adrian Krainer, former postdoc Wai Kit Ma, and Dillon ...
Feb 3, 2022
0
10
Diseases, Conditions, Syndromes
Researchers develop tools to measure skeletal muscle function in patients with mitochondrial disease
Researchers at Children's Hospital of Philadelphia (CHOP) successfully developed and validated a new outcome measure to monitor disease severity and progression in patients with impaired skeletal muscle function caused by ...
Sep 1, 2021
0
8
Medical economics
New gene therapies may soon treat dozens of rare diseases, but expense may limit availability for many
Zolgensma—which treats spinal muscular atrophy, a rare genetic disease that damages nerve cells, leading to muscle decay—is currently the most expensive drug in the world. A one-time treatment of the life-saving drug ...
Sep 1, 2021
0
16
Genetics
Testing and treating newborns for spinal muscular atrophy: Saving lives and healthcare costs
Gene therapy for spinal muscular atrophy might have a high up-front price tag. But by screening and treating infants early, the therapy can save both lives and money in the long term.
Aug 3, 2021
0
10
Medications
The 'most expensive drug in the world', how it works and the devastating disease it treats
Babies born with the rare, inherited motor neuron disease spinal muscular atrophy are, without treatment, unlikely to reach their second birthday. When, as a researcher in the 1990s, I became aware of the disease there were ...
Jul 20, 2021
0
3
Genetics
New components for antisense gene therapy show promise in treating spinal muscular atrophy
Skoltech researchers and their colleagues from Russia and the UK investigated the safety and efficacy of new chemistry in antisense oligonucleotides used to treat spinal muscular atrophy (SMA), a debilitating genetic disease. ...
Jun 29, 2021
0
3
Genetics
New study expands evaluation of gene therapy for spinal muscular atrophy
The rarity of spinal muscular atrophy (SMA) means that promising new treatments may be tested in only a limited spectrum of patients before approval. Investigators evaluated a newly approved drug, onasemnogene abeparvovec, ...
Apr 6, 2021
0
6
Neuroscience
Researchers discover how the brain 're-wires' after disease
Trinity researchers are studying how the brain re-wires itself in neurological disease. The team is building treatments for today's more common global conditions like motor neurone disease (MND/ALS) and spinal muscular ...
Nov 17, 2020
0
30
