Muscular Atrophy News and Research

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Uncovering the pathway behind ALS neurodegeneration

Approximately 5,000 people in the U.S. develop amyotrophic lateral sclerosis (ALS) each year. On average, they survive for only two to five years after being diagnosed, according to the Centers for Disease Control and Prevention.

8 Nov 2024

Scientists develop selective therapy for tau tangles

Scientists have developed new potential therapies that selectively remove aggregated tau proteins, which are associated with Alzheimer's disease, and improve symptoms of neurodegeneration in mice.

14 Sep 2024

New dye technique allows for transparent skin imaging in live mice

Seeing what's going on inside a body is never easy. While technologies like CT scans, X-rays, MRIs, and microscopy can provide insights, the images are rarely completely clear and can come with side effects like radiation exposure.

5 Sep 2024

Alternative splicing may influence gene expression more than protein diversity

Alternative splicing is a genetic process where different segments of genes are removed, and the remaining pieces are joined together during transcription to messenger RNA (mRNA).

2 Sep 2024

Robotic gait training improves mobility in patients with spinal and bulbar muscular atrophy

Spinal and bulbar muscular atrophy (SBMA) is a rare X-linked neuromuscular disorder that results from an abnormal expansion of gene sequences in the androgen receptor gene.

22 Aug 2024

Scientists explore early life factors contributing to SMA

Spinal muscular atrophy (SMA) is a severe neurological disease for which there is presently no cure, although current therapies can alleviate symptoms. In the search for better treatment options, scientists at DZNE and the Dresden University of Technology are now drawing attention to previously unnoticed abnormalities in embryonic development. They base their argument on studies of so-called organoids: Laboratory-grown tissue cultures that can reconstruct disease processes. Their findings are published in the journal Cell Reports Medicine.

27 Jul 2024

Understanding Global Harmonization of Regulatory CMC within Veterinary Medicine with Tammy Newbold

In this interview, News Med talks to Tammy Newbold about Global Harmonization of Regulatory CMC within Veterinary Medicine.

From DSI, a PLG Company 26 Jul 2024

Spinal Muscular Atrophy patients are at higher risk of developing fatty liver disease

An international study has discovered that people living with the neurodegenerative condition Spinal Muscular Atrophy (SMA) are at higher risk of developing fatty liver disease, suggesting that SMA patients may face additional health problems over time.

17 Jul 2024

Comparative study on preventative SMA therapies shows mixed outcomes

In a first-of-its-kind study, researchers compared the efficacy of preventative therapy for spinal muscular atrophy (SMA) between two well-matched study groups, using either gene therapy (onasemnogene abeparvovec) alone or in combination with risdiplam (oral medication) or nusinersen (intrathecal injection) administered before apparent signs of disease emerged.

25 Jun 2024

CSHL team identifies crucial protein interactions in RNA splicing process

Bodybuilders and cellular mechanisms agree generating protein is a heavy lift. To complete the task, cells rely on complexes called spliceosomes.

10 Jun 2024

Novel gene-delivery vehicle shows promise for treating brain disorders

In an important step toward more effective gene therapies for brain diseases, researchers from the Broad Institute of MIT and Harvard have engineered a gene-delivery vehicle that uses a human protein to efficiently cross the blood-brain barrier and deliver a disease-relevant gene to the brain in mice expressing the human protein.

16 May 2024

Real-world data validates gene therapy for spinal muscular atrophy

Findings from a new study in the Journal of Neuromuscular Diseases, published by IOS Press, demonstrate the effectiveness of disease-modifying treatments (DMTs) in infants with spinal muscular atrophy (SMA).

15 Apr 2024

Discovery of a fundamental biological mechanism opens path for novel therapies

The lab of Yongchao C. Ma, PhD, at Stanley Manne Children's Research Institute at Ann & Robert H. Lurie Children's Hospital of Chicago discovered a fundamental biological mechanism that could lead to new treatments for neurological diseases, such as spinal muscular atrophy (SMA) and autism, as well as different cancers.

4 Apr 2024