Progressive Muscular Atrophy (PMA)

Treatments available for progressive muscular atrophy (PMA) are limited, making it essential to find new and effective therapies for the continuous breakdown of motor neurons and muscle deterioration linked with the condition. Protheragen is positioned optimally to assist you in your PMA therapy research and guide you to commercialization because of our extensive knowledge regarding PMA therapy development which places us at the forefront of offering tailored solutions and holistic assistance.

Introduction to Progressive Muscular Atrophy (PMA)

Progressive muscular atrophy (PMA) is a rare type of motor neuron disease which occurs sporadically. It manifests through the degeneration of lower motor neurons (LMN), resulting in asymmetric muscle weakness, atrophy, fasciculations, and reduced reflexes without any upper motor neuron involvement. It is distinguished from amyotrophic lateral sclerosis (ALS) due to its slower rate of progression and pure LMN pathology. PMA usually presents with distal-onset weakness first noticed in the hands or legs.

Fig.1 Primary lateral sclerosis (PLS) and progressive muscular atrophy (PMA) patients displayed similar cognitive and behavioural impairment profile than ALS patients. (Dr José Manuel Matamala., 2019)

Pathogenesis of Progressive Muscular Atrophy (PMA)

Progressive muscular atrophy (PMA) develops due to the selective degeneration of lower motor neurons (LMNs) in the spinal cord and brainstem, driven by various factors such as protein misfolding (e.g., TDP-43 and SOD1 aggregation), mitochondrial abnormalities, or problems with axonal transport. PMA is very different from ALS in that it only shows lower motor neuron pathology. However, 30-50% of PMA cases show TDP-43 inclusions which suggests some overlap with ALS-spectrum disorders.

Fig.2 Genes implicated in various motor neuron diseases (MNDs). (Antonakoudis A, et al., 2025)

Therapeutic Development for Progressive Muscular Atrophy (PMA)

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

Recognizing the complexity of diagnosing and treating progressive muscular atrophy (PMA), Protheragen is committed to building a team of experts to provide cutting-edge diagnostic and therapeutic development solutions. Our commitment lies in providing a variety of customized therapy development services to meet the diverse research needs of our customers. We also excel in generating precise disease models, including in vitro models, animal models and special blood-brain barrier (BBB) models, which are carefully designed to reproduce the unique characteristics of PMA.

Therapeutic Development Services

By Molecule Types

At Protheragen, we overcome bottlenecks in progressive muscular atrophy (PMA) drug development through comprehensive and multimodal drug development platforms to accelerate therapies from discovery to clinical translation.

• Small Molecule Drug Development
• Cell Therapy Development
• Gene Therapy Development

• Therapeutic Antibody Development
• Therapeutic Peptide Development
• Therapeutic Protein Development

By Mechanism of Action

Protheragen specializes in developing customized neurotherapeutic drugs targeting the underlying pathological mechanisms of progressive muscular atrophy (PMA), leading to groundbreaking treatment solutions.

• Neuroprotective Agent Development
• Glutamate Modulator Development
• Anti-neuroinflammatory Drug Development
• Antioxidant Development

• Mitochondria-targeted Drug Development
• Protein Misfolding Inhibitor Development
• Protein Aggregation Inhibitor Development
• Excitotoxicity Inhibitor Development

Disease Model Development Services

In Vitro Model
Development

• Patient-Derived Cell Model
• Neuronal Cell Model
• Glial Cell Model
• Co-Culture Model
• Brain Organoid
• Spinal Cord Organoid

Microfluidic Model
Development

• Neurovascular Unit (NVU) Model
• Axon Degeneration Model
• Neuroinflammation Model
• Neuromuscular Junction (NMJ) Model
• Synaptic Plasticity Model
• Organoid-on-a-Chip Model

Animal Model
Development

• Wobbler Mouse: This is a well-established model exhibiting progressive muscle weakness and atrophy, resembling human PMA.
• SOD1G93A Mouse: This model, often used for ALS, can also display features relevant to PMA due to the involvement of lower 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

• Antonakoudis A, Kyriakoudi S A, Chatzi D, et al. Genetic Basis of Motor Neuron Diseases: Insights, Clinical Management, and Future Directions[J]. International Journal of Molecular Sciences, 2025, 26(10): 4904.

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