Nucleic Acid and Gene Therapies in Neuromuscular Disorders: Analysis of Epidemiology, Pipeline Therapies, and Key Companies Working in the market

November 08 18:20 2022
Nucleic Acid and Gene Therapies in Neuromuscular Disorders: Analysis of Epidemiology, Pipeline Therapies, and Key Companies Working in the market
DelveInsight Business Research LLP
DelveInsight’s “Nucleic Acid and Gene Therapies in Neuromuscular Disorders Market Insights, Epidemiology, and Market Forecast-2032” report delivers an in-depth understanding of the Allergic Rhinitis, historical and forecasted epidemiology as well as the Nucleic Acid and Gene Therapies in Neuromuscular disorder market trends in the United States, EU5 (Germany, Spain, Italy, France, and United Kingdom) and Japan.

DelveInsight’s “Nucleic Acid and Gene Therapies in Neuromuscular Disorders Market Insights, Epidemiology, and Market Forecast-2032” report delivers an in-depth understanding of the Allergic Rhinitis, historical and forecasted epidemiology as well as the Nucleic Acid and Gene Therapies in Neuromuscular disorder market trends in the United States, EU5 (Germany, Spain, Italy, France, and United Kingdom) and Japan.

 

The Nucleic Acid and Gene Therapies in Neuromuscular Disorders market report provides current treatment practices, emerging drugs, Allergic Rhinitis market share of the individual therapies, current and forecasted Nucleic Acid and Gene Therapies in Neuromuscular Disorders market Size from 2019 to 2032 segmented by seven major markets. The Report also covers current Allergic Rhinitis treatment practice/algorithm, market drivers, market barriers and unmet medical needs to curate the best of the opportunities and assesses the underlying potential of the Nucleic Acid and Gene Therapies in the neuromuscular disorders market.

Some of the key facts of the Nucleic Acid and Gene Therapies in Neuromuscular Disorders Market Report:

  •  In France, a study by Foulon S. et al. (2017), found that the overall prevalence rate of MS was 155.6/100,000 inhabitants after the standardization of the 2013-European population. We observed a prevalence gradient with a higher prevalence (190-200 per 100,000) in North-Eastern France and a lower prevalence in Southern and Western France (126-140).

  •  Centers for Disease Control and Prevention (CDC) published data stating that the traditional registry methods for estimating the prevalence of Amyotrophic lateral sclerosis calculated the age-adjusted prevalence at 5.5/per 100,000 persons.

  • A study conducted by Brown C.A. (2021), the pooled prevalence rate (per 100,000) of amyotrophic lateral sclerosis (ALS) was found to be 6.22 in the European countries.

 

Nucleic Acid and Gene Therapies in Neuromuscular Disorders Overview

Neuromuscular disorders (NMD) include a wide range of diseases that affect the peripheral nervous system, which consists of all the motor and sensory nerves that connect the brain and spinal cord to the rest of the body. When the nerves are damaged, the communication between the nerves and muscles becomes disrupted which then results in muscle weakness, wasting, and loss of function. The nerves stay in touch with the muscles through the release of neurotransmitters at the neuromuscular junction (the space between a nerve cell and a muscle fiber. The neuromuscular disorders can damage the nerve itself or the neuromuscular junction, where the signal is transmitted from a nerve to a muscle.There are many different classes of diseases involved in neuromuscular disorders which may include Muscular dystrophies, myopathies, motor neuron diseases, ion channel diseases, mitochondrial diseases, neuromuscular diseases, and peripheral nerve diseases.

Nucleic Acid and Gene Therapies in Neuromuscular Disorders Market Outlook 

The neuromuscular disorders comprise a number (approx. 300) of inherited or acquired disorders that primarily affect one or more components of the motor unit (motoneuron and the group of skeletal muscle fibers each motor neuron activates). The treatment of neuromuscular  diseases faces numerous challenges:  the need to correct and preserve the totality of the target tissue (mainly the muscle, which represents half of the total mass of the organism, and the peripheral nervous system with specific bioavailability issues) in a durable manner;  the fact that they are serious disorders beginning in infancy and evolving towards a progressive disappearance of functional tissue; and  the considerable variety of genetic anomalies that are unique for each patient

 

Nucleic Acid and Gene Therapies in Neuromuscular Disorders- Key Companies

  •  Sarepta Therapeutics

  •  NS Pharma

  •  Inmunotek 

  •  Novartis, Biogen, 

  • Ionis Pharmaceuticals,

  •  Helix Smith

  •  Novartis 

  •  ALK Abello

  •  Stallergenes 

  •  Pfizer and many others

Nucleic Acid and Gene Therapies in Neuromuscular Disorders – Therapies

  •  SRP-9001 

  •  PF-06939926 (fordadistrogene movaparvovec)

  •  Branaplam (LMI070 and NVS-SM1)

  •  ION363 is an investigational antisense medicine

  • Tofersen (BIIB067) is an antisense drug and many others 

 

Table of Contents

  •  Key Insights 

  •  Report Introduction

  •  Executive Summary of Nucleic Acid and Gene Therapy

  •  Disease Background and Overview

  •  Epidemiology and patient population

  •  Nucleic Acid and Gene Therapy  Emerging Therapies

  •  Allergic Rhinitis Market Outlook

  • Market Access and Reimbursement of Therapies

  •  Appendix

  •  Nucleic Acid and Gene Therapy Report Methodology

  •  DelveInsight Capabilities

  •  Disclaimer

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