„Prelude“ to Neuromuscular Disease SMA May Offer Chances for Better Treatment

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”.

Background Research:

Spinal Muscular Atrophy (SMA) is a genetic disease affecting the part of the nervous system that controls voluntary muscle movement. Most of the nerve cells that control muscles are located in the spinal cord, which is why it is called „spinal“ muscular atrophy.

The severity of SMA and the manner in which it affects people can vary widely. In some cases, infants diagnosed with a severe type of SMA may not survive past early childhood due to respiratory failure. On another hand, children with less severe types can learn to sit up, walk and even run but will experience progressive muscle wasting and weakness over time.

Currently, there are four known types (SMA1-4) depending upon age at onset and maximally achieved motor skill.
Approximately one in every 10,000 babies is born with SMA. The parents usually have no history of the disease.

Possible FAQ for Article:

1. What is Spinal Muscular Atrophy?
Answer: Spinal muscular atrophy (SMA) is a genetic disorder that affects voluntary muscle movement because most motor neurons controlling these movements are located within the spinal cord area.

2.What cause Spinal Muscular Atrophy?
Answer: SMA is caused by mutation or deletion on chromosome 5 leads to deficiency/absence of survival motor neuron protein critical for nerve function

3.How many types does Spinal Muscular Dystrophy has?
Answer: There are four main types recognized based on onset age & overall impact; Type I(SMA1), II(SMA2), III(SMA3), IV(SKA4)

4.What makes this research different from previous studies?
Answer: The research team focused more on embryonic developmental abnormalities instead focusing only on treating symptoms like previous studies did. This will pave way for development new treatment strategies targeting underlying abnormal process rather than end-stage completed processes which could be more effective.

5.What’s an organoid, and how does this play into the research?
Answer: Organoids are miniaturized and simplified 3D structures resembling real organs. The organoids allow scientists to recreate diseases in lab making it easier understand disease processes closely

6.What does this mean for people living with SMA?
Answer: If the abnormalities detected early before massive damage occurs,it could be corrected through medication-thus mitigating or even slowing down production of faulty proteins or enhancing production of normal protein whichever seems viable based on particular context thus improving their quality of life.

7.What is DZNE?
Answer:DZNE is abbreviation for German Center Neurodegenerative Diseases, a research institution dedicated to study causes behind neurodegenerative diseases & to developing effective therapies.

8.Is there complete cure for SMA till now?
Answer: No. Till present day there’s no complete cure but current treatment modalities can alleviate symptoms and discomfort by correcting supportive care issues that come with SMA.

9.Where can I find more details about the research publication?
Link – http://idw-online.de/de/news837533

Originamitteilung:

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”.

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