Centronuclear Myopathy - Pipeline Insight, 2024

This “Centronuclear Myopathy- Pipeline Insight, 2024” report provides comprehensive insights about 5+ companies and 5+ pipeline drugs in Centronuclear Myopathy pipeline landscape. It covers the pipeline drug profiles, including clinical and nonclinical stage products. It also covers the therapeutics assessment by product type, stage, route of administration, and molecule type. It further highlights the inactive pipeline products in this space.

Geography Covered

• Global coverage

Centronuclear Myopathy: Understanding

Centronuclear Myopathy: Overview

Centronuclear myopathies are a group of muscle diseases that are considered part of a larger family of muscle diseases known as congenital myopathies, a group of genetic muscle disorder that are evident at or around the time of birth. The centronuclear myopathies are a group of rare inherited neuromuscular conditions that cause muscle weakness and hypotonia (lack of muscle tone). The symptoms range from severely disabling conditions that are diagnosed at birth to relatively mild ones with onset in adulthood. There are three forms: X-linked, autosomal recessive and autosomal dominant. These classifications are based on the way in which the conditions are passed down through families. X-linked centronuclear myopathy is also known as myotubular myopathy. Usually muscle fibres have their nucleus (control centre) located at the edge but in people with centronucular myopathy the nucleus is in the centre of the muscle fibre. People with centronuclear myopathy begin experiencing muscle weakness at any time from birth to early adulthood. The muscle weakness slowly worsens over time and can lead to delayed development of motor skills, such as crawling or walking; muscle pain during exercise; and difficulty walking. Some affected individuals may need wheelchair assistance as the muscles atrophy and weakness becomes more severe. In rare instances, the muscle weakness improves over time. Some people with centronuclear myopathy experience mild to severe breathing problems related to the weakness of muscles needed for breathing. People with centronuclear myopathy may have droopy eyelids (ptosis) and weakness in other facial muscles, including the muscles that control eye movement. CNM should be suspected in newborns with hypotonia and muscle weakness and older children or adults with weakness in the arms and legs. A diagnosis is based upon identification of additional characteristic symptoms, a detailed patient and family history, a thorough clinical evaluation, and a variety of specialized tests. A muscle biopsy may be performed to aid in obtaining a diagnosis. A biopsy involves surgical removal of a small sample of affected muscle tissue and examining the sample under a microscope. Specialized imaging techniques such as a muscle MRI (magnetic resonance imaging) may be used to inform the choice of genetic testing in different forms of CNM, particularly in DNM2- and RYR1-related CNM. An MRI uses a magnetic field to produce cross-sectional images of particular organs and bodily tissues such as skeletal muscle tissue. The treatment of CNM is directed toward the specific symptoms that are apparent in each individual. Treatment may require the coordinated efforts of a team of specialists with expertise in treating neuromuscular disorders. Pediatricians, pulmonologists, neurologists, orthopedists, eye specialists, dental specialists, and other healthcare professionals may need to systematically and comprehensively plan an affected child’s treatment. Genetic counseling will be of benefit for affected individuals and their families.

Centronuclear Myopathy- Pipeline Insight, 2024 report outlays comprehensive insights of present scenario and growth prospects across the indication. A detailed picture of the Centronuclear Myopathy pipeline landscape is provided which includes the disease overview and Centronuclear Myopathy treatment guidelines. The assessment part of the report embraces, in depth Centronuclear Myopathy commercial assessment and clinical assessment of the pipeline products under development. In the report, detailed description of the drug is given which includes mechanism of action of the drug, clinical studies, NDA approvals (if any), and product development activities comprising the technology, Centronuclear Myopathy collaborations, licensing, mergers and acquisition, funding, designations and other product related details.

Report Highlights

The companies and academics are working to assess challenges and seek opportunities that could influence Centronuclear Myopathy R&D. The therapies under development are focused on novel approaches to treat/improve Centronuclear Myopathy.

Centronuclear Myopathy Emerging Drugs Chapters

This segment of the Centronuclear Myopathy report encloses its detailed analysis of various drugs in different stages of clinical development, including phase II, I, preclinical and Discovery. It also helps to understand clinical trial details, expressive pharmacological action, agreements and collaborations, and the latest news and press releases.

Centronuclear Myopathy Emerging Drugs

DYN101: Dynacure IONIS-DNM2-2.5Rx (DYN101) is an investigational antisense medicine designed to reduce the production of Dynamin 2 (DNM2) protein for the treatment of centronuclear myopathy (CNM). Preclinical studies have shown that DYN101 has the potential to be disease modifying in CNM, with preclinical activity observed in animal models of XLCNM and ADCNM. Prevention and reversion of the disease was observed with a dose-dependent improvement in whole body strength and mice survival. The development plan for DYN101 was designed to be broad and it is the only known program investigating a treatment for the majority of CNM patients, comprised of those who have XLCNM or ADCNM. DYN101 is currently under clinical investigation in several European countries. DYN101 has been granted Orphan Drug designations by the FDA and EMA and rare pediatric disease designation by theFDA.

Centronuclear Myopathy: Therapeutic Assessment

This segment of the report provides insights about the different Centronuclear Myopathy drugs segregated based on following parameters that define the scope of the report, such as:

Major Players in Centronuclear Myopathy

There are approx. 5+ key companies which are developing the therapies for Centronuclear Myopathy. The companies which have their Centronuclear Myopathy drug candidates in the most advanced stage, i.e. phase I /II include, Dynacure.

Phases

This report covers around 5+ products under different phases of clinical development like

• Late stage products (Phase III)
• Mid-stage products (Phase II)
• Early-stage product (Phase I) along with the details of
• Pre-clinical and Discovery stage candidates
• Discontinued & Inactive candidates

Route of Administration

Centronuclear Myopathy pipeline report provides the therapeutic assessment of the pipeline drugs by the Route of Administration. Products have been categorized under various ROAs such as

• Intra-articular
• Intraocular
• Intrathecal
• Intravenous
• Ophthalmic
• Oral
• Parenteral
• Subcutaneous
• Topical
• Transdermal

Molecule Type

Products have been categorized under various Molecule types such as

• Oligonucleotide
• Peptide
• Small molecule

Product Type

Drugs have been categorized under various product types like Mono, Combination and Mono/Combination.

Centronuclear Myopathy: Pipeline Development Activities

The report provides insights into different therapeutic candidates in phase II, I, preclinical and discovery stage. It also analyses Centronuclear Myopathy therapeutic drugs key players involved in developing key drugs.

Pipeline Development Activities

The report covers the detailed information of collaborations, acquisition and merger, licensing along with a thorough therapeutic assessment of emerging Centronuclear Myopathy drugs.

Centronuclear Myopathy Report Insights

• Centronuclear Myopathy Pipeline Analysis
• Therapeutic Assessment
• Unmet Needs
• Impact of Drugs

Centronuclear Myopathy Report Assessment

• Pipeline Product Profiles
• Therapeutic Assessment
• Pipeline Assessment
• Inactive drugs assessment
• Unmet Needs

Key Questions

Current Treatment Scenario and Emerging Therapies:

• How many companies are developing Centronuclear Myopathy drugs?
• How many Centronuclear Myopathy drugs are developed by each company?
• How many emerging drugs are in mid-stage, and late-stage of development for the treatment of Centronuclear Myopathy?
• What are the key collaborations (Industry-Industry, Industry-Academia), Mergers and acquisitions, licensing activities related to the Centronuclear Myopathy therapeutics?
• What are the recent trends, drug types and novel technologies developed to overcome the limitation of existing therapies?
• What are the clinical studies going on for Centronuclear Myopathy and their status?
• What are the key designations that have been granted to the emerging drugs?

Key Players

• Dynacure
• Astellas Gene Therapies
• ARMGO Pharma

Key Products

• AT-132
• DYN101
• ARM210

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