Emerging Pipeline Therapeutics Across 6 Rare Neuromuscular Diseases: Strategic Partnering and New Product Development will Enable Better Biologics for Personalized and Targeted Treatment – ResearchAndMarkets.com

DUBLIN–(BUSINESS WIRE)–The “Emerging Therapeutics for Rare Neuromuscular Diseases: Pipeline Analysis” report has been added to ResearchAndMarkets.com’s offering.

Biologics presents a personalized and targeted method to treat NMDs and is a promising treatment class. An overview of the global clinical trial landscape for emerging Phase 1, 2, and 3 treatments revealed that Amyotrophic Lateral Sclerosis (ALS), Myasthenia Gravis (MG), Duchenne Muscular Dystrophy (DMD), Chronic Inflammatory Demyelinating Polyradiculoneuropathy (CIDP), Diabetic Neuropathy (DN), and Spinal Muscular Atrophy (SMA) are the key hotspots for clinical studies. These indications are likely to witness new drug developments.

This research, “Emerging Therapeutics for Rare Neuromuscular Diseases: A Pipeline Analysis” explores emerging biologics across these six NMDs.

Muscle weakness, cramps, and impaired musculoskeletal functions that occur due to rare defects in nerve and muscle cells characterize neuromuscular disorders (NMDs). The disorders are largely classified as motor neuron diseases, hereditary ataxias, peripheral nerve disorders, neuromuscular junction transmission disorders, and myopathies.

NMDs present significant clinical challenges because of disease heterogeneity and rarity of occurrence. The lack of measurable early disease markers and clinical outcomes further complicates the management of such disorders.

Key biologics emerging for these rare NMDs include stem cell therapies, other cell-based treatments, gene therapies, RNA therapeutics, immunotherapies, and other protein/peptide-based treatments. There were 3 recent US FDA approvals for NMD RNA therapies: Nusinersen for SMA and Eteplirsen for DMD in 2016, and Golodirsen for DMD in 2019.

Furthermore, Sarepta’s Casimersen for DMD and argenx’s efgartigimod for MG were approved as recently as 2021. Biologics have opened doors for ground-breaking disease-modifying treatments and will transform the quality of life for patients across the world.

While biologics may appear to cost more than small molecule treatments, they provide a personalized and, in some cases, the only treatment option. They can also be less expensive than lifetime treatment costs for certain rare NMDs. For example, Zolgensma’s $2.1 million price tag (approximate) is still lower than the standard medical costs incurred for SMA patients in a lifetime. Furthermore, the evolving payer landscape is likely to get more standardized in the coming years to offer greater access to biologicals.

Therefore, biologics are suitably poised to transform the rare NMD landscape with personalized and targeted treatment strategies and will provide life-saving treatment options for fatal neuromuscular conditions.

Key Points Discussed

What are the key emerging biologics for NMDs with high clinical activity?

What are the key drivers or challenges for biologics development across NMDs?

How do venture funding and patent landscapes look for NMD biologics?

Who are the key industry participants developing biologics for NMDs?

What are the clinical trends emerging across each biologic category for NMDs?

Which biologic categories provide promising growth opportunities for NMD management?

Key Topics Covered:

Strategic Imperatives

Factors Creating Pressure on Growth

The Impact of the Top 3 Strategic Imperatives on Rare Neuromuscular Disease Management

Growth Opportunities Fuel the Growth Pipeline Engine

Research Methodology

Growth Opportunity Analysis

Scope of Analysis

Segmentation

Predominant Emerging Biologic Classes Across NMDs

Growth Drivers

Growth Restraints

Growth Environment

Sharp Growth in Investor Interest for Rare NMD Treatments

Snapshot of Venture-backed Companies that Enable Biologics Progress

Biologic Patents for NMDs

Patent Landscape – Biologics for NMDs

Top Participants

ALS: Biologics Pipeline Analysis

Biological Therapies for ALS

ALS Cell Therapies, 2022

ALS Oligonucleotide Therapies, 2022

ALS Protein Therapies, 2022

Highlights of Recent Biologics Studies for ALS Treatment

Exploring New Therapy Paradigms for ALS Treatment

MG: Biologics Pipeline Analysis

Upcoming Biologics for MG Treatment

Protein Therapies for MG

Clinical Trial Progress in Biologics* for MG

Dominant MG Biologics

DMD: Biologics Pipeline Analysis

Gene Therapies in the Emerging DMD Biologics Landscape

Exon-Skipping Therapies for DMD

Microdystrophin Gene Therapies for DMD

Oligonucleotide Therapies to Lead DMD Management

SMA: Biologics Pipeline Analysis

Approved and Emerging Biologics Classes for SMA

Biologics Transforming the Clinical Landscape for SMA

SMA Management Through Groundbreaking Therapies

Novartis’ Zolgensma: A Clinical Breakthrough for SMA

DN & CIDP: Biologics Pipeline Analysis

Biologics for DN Treatment

Biologics for Treatment Personalization in DN

Emerging Biologics for CIDP Treatment

Immunoglobulins for CIDP Treatments

Growth Opportunity Universe

Growth Opportunity 1: RNA Therapeutics

Growth Opportunity 2: Cell and Gene Therapeutics

Growth Opportunity 3: Protein Therapeutics

For more information about this report visit https://www.researchandmarkets.com/r/sgh4df

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