Flail Arm Syndrome (FAS)
To address the progressive effects of flail arm syndrome (FAS), targeted treatments that can effectively halt or reverse neuronal degeneration are urgently needed. At Protheragen, we focus on developing novel therapeutics and building accurate animal models to accelerate preclinical studies of potential therapies for FAS. Our expertise ensures that your research receives the most reliable and relevant support, accelerating your drug development journey.
Introduction to Flail Arm Syndrome (FAS)
Flail arm syndrome (FAS), or brachial amyotrophic diplegia and man-in-the-barrel syndrome, is a lesser-known disorder concerning the nervous system defined by increasingly more pronounced muscle degeneration and weakness around the upper arms and shoulders. FAS is viewed as a subtype of amyotrophic lateral sclerosis (ALS) but it is a lower motor neuron dominant variant. Unlike typical ALS cases, FAS has a much slower rate of progression and life expectancy averages around 5 to 10 years, mostly seen in men.
Fig.1 Pattern of motor involvement across the amyotrophic lateral sclerosis (ALS) clinical phenotypes. (Dharmadasa T., 2021)
Pathogenesis of Flail Arm Syndrome (FAS)
The development of flail arm syndrome (FAS) is associated with the selective degeneration of cervical lower motor neurons. This form of selective degeneration is driven by certain genetic changes, particularly expansions in C9ORF72 hexanucleotide sequences and variants in SOD1, along with some degree of mitochondrial dysfunction and TDP-43 proteinopathy. These changes interfere with RNA metabolism as well as axonal transport, resulting in considerable loss of motor neurons in the cervical region of the spinal cord while upper motor neurons are preserved to a significant extent. Enhanced neuroinflammation due to activated microglia and increased pro-inflammatory cytokines compounds the injury to nerve cells and results in muscle weakness and atrophy.
Therapeutic Development for Flail Arm Syndrome (FAS)
| Drug Names | Mechanism of Action | Targets | Research Phase |
| L-Carnitine | Facilitates the transport of fatty acids into the mitochondria for energy production, potentially protecting neuronal cells. | Mitochondrial membrane | Phase II/III |
| Riluzole | Inhibits glutamate release, inactivates voltage-dependent sodium channels, and interferes with intracellular events following excitatory amino acid receptor binding, thereby potentially reducing neurotoxicity. | Glutamate receptors, voltage-gated sodium channels | Early Research |
Disclaimer: Protheragen focuses on providing preclinical research services. This table is for information exchange purposes only. This table is not a treatment plan recommendation. For guidance on treatment options, please visit a regular hospital.
Our Services
Protheragen focuses on offering modern diagnostic and therapeutic development services for rare lower motor neuron diseases (LMND) such as flail arm syndrome (FAS). Our priorities include designing sophisticated models of the disease to refine understanding of FAS's pathogenesis, alongside utilizing novel blood-brain barrier (BBB) models to improve the delivery systems and effectiveness of therapeutic agents on meeting synergistic outcomes. Through these methods, we strive to advance integrated diagnostic mechanisms and multi-vector therapeutics aligned with the precise pathobiology of FAS.
Therapeutic Development Services
By Molecule Types
To address flail arm syndrome (FAS), Protheragen strives to create small molecule drugs, gene therapies, and biological agents by targeting intricate pathways involved in FAS's core mechanisms.
By Mechanism of Action
Protheragen has specialized capabilities to develop medicines such as neuroprotective medicines, anti-neuritis drugs, and other specialty medicines that diminish inflammation in the nervous system while also protecting neurons from degeneration.
Disease Model Development Services
In Vitro Model
Development
Development
- SOD1 Transgenic Model: Overexpression of mutant SOD1 to mimic motor neuron degeneration.
- Cervical Spinal Cord Ischemic Injury Model: Bilateral photothrombosis or vascular occlusion targeting cervical motor neurons.
Focusing on preclinical research, Protheragen offers comprehensive pharmacodynamic (PD), pharmacokinetic (PK), and toxicology study services to support the development and regulatory approval of potential therapies. If you are interested in our services, please feel free to contact us for more details and quotation information of related services.
Reference
- Dharmadasa T. Cortical excitability across the ALS clinical motor phenotypes[J]. Brain Sciences, 2021, 11(6): 715.