DelveInsight’s, “Spinal Muscular Atrophy Pipeline Insight 2023” report provides comprehensive insights about 18+ companies and 20+ pipeline drugs in the Spinal muscular atrophy pipeline landscape. It covers the Spinal Muscular Atrophy pipeline drug profiles, including clinical and nonclinical stage products. It also covers the Spinal Muscular Atrophy pipeline therapeutics assessment by product type, stage, route of administration, and molecule type. It further highlights the inactive pipeline products in this space.
Key Takeaways from the Spinal Muscular Atrophy Pipeline Report
- DelveInsight’s Spinal Muscular Atrophy pipeline report depicts a robust space with 18+ active players working to develop 20+ pipeline therapies for Spinal Muscular Atrophy treatment.
- The leading companies working in the Spinal Muscular Atrophy Market include Scholar Rock, Biogen, Astellas Pharma, Alcyone Therapeutics, AndroScience Corporation, Hanugen Theraputics, Voyager Therapeutics, Hoffmann-La Roche, Catalyst Pharmaceuticals, NMD Pharma, Biohaven Pharmaceuticals, CANbridge Pharmaceuticals Inc., Aurimed Pharma, Exicure, Amylon Therapeutics, Amniotics, and others.
- Promising Spinal Muscular Atrophy Pipeline Therapies in the various stages of development include Reldesemtiv 150 mg, Zolgensma, Branaplam, EXG001-307 injection, OAV101, and others.
- September 2023: Genentech Inc. announced a study of phase 4 clinical trials for Risdiplam. A multi-center, longitudinal, prospective, non-comparative study to investigate the long-term safety and effectiveness of risdiplam, prescribed based on clinician judgment as per the Evrysdi® U.S. Package Insert (USPI) in adult and pediatric participants with spinal muscular atrophy (SMA). In this study, participants will be followed for up to 5 years from enrollment or until withdrawal of consent, loss to follow-up, or death. Participants who discontinue risdiplam may still remain in the study, if they agree to continue participating in the follow-up assessments.
- September 2023: NMD Pharma A/S announced a study of phase 2 clinical trials for NMD670. The purpose of this study is to evaluate the efficacy, safety, tolerability and pharmacokinetics of NMD670 in the treatment of ambulatory adults with spinal muscular atrophy type 3.
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In the Spinal Muscular Atrophy pipeline 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, Spinal muscular atrophy collaborations, licensing, mergers and acquisition, funding, designations and other product related details.
Spinal Muscular Atrophy Overview
Spinal muscular atrophy (SMA) is a genetic disease affecting the central nervous system, peripheral nervous system, and voluntary muscle movement (skeletal muscle). Most of the nerve cells that control muscles are located in the spinal cord, which accounts for the word spinal in the name of the disease.
Find out more about Spinal Muscular Atrophy Therapeutics Assessment @ Spinal Muscular Atrophy Preclinical and Discovery Stage Products
Spinal Muscular Atrophy Emerging Drugs Profile
- Apitegromab: Scholar Rock
- ACTX-401: Alcyone Therapeutics
Spinal Muscular Atrophy Pipeline Therapeutics
There are approx. 18+ key companies which are developing the Spinal Muscular Atrophy therapies. The Spinal Muscular Atrophy companies which have their Spinal muscular atrophy drug candidates in the most advanced stage, i.e. phase III include, Scholar Rock.
Learn more about the emerging Spinal Muscular Atrophy Pipeline Therapies @ Spinal Muscular Atrophy Clinical Trials Assessment
Scope of the Spinal Muscular Atrophy Pipeline Report
- Coverage- Global
- Spinal Muscular Atrophy Therapeutic Assessment by Product Type: Mono, Combination, Mono/Combination
- Spinal Muscular Atrophy Therapeutic Assessment by Clinical Stages: Discovery, Pre-clinical, Phase I, Phase II, Phase III
- Spinal Muscular Atrophy Companies- Scholar Rock, Biogen, Astellas Pharma, Alcyone Therapeutics, AndroScience Corporation, Hanugen Theraputics, Voyager Therapeutics, Hoffmann-La Roche, Catalyst Pharmaceuticals, NMD Pharma, Biohaven Pharmaceuticals, CANbridge Pharmaceuticals Inc., Aurimed Pharma, Exicure, Amylon Therapeutics, Amniotics, and others.
- Spinal Muscular Atrophy Pipeline Therapies- Reldesemtiv 150 mg, Zolgensma, Branaplam, EXG001-307 injection, OAV101, and others.
Dive deep into rich insights for new drugs for Spinal Muscular Atrophy Treatment, Visit @ Spinal Muscular Atrophy Market Drivers and Barriers, and Future Perspective
Table of Content
- Introduction
- Executive Summary
- Spinal muscular atrophy: Overview
- Pipeline Therapeutics
- Therapeutic Assessment
- Spinal muscular atrophy – DelveInsight’s Analytical Perspective
- Late Stage Products (Phase III)
- Apitegromab: Scholar Rock
- Drug profiles in the detailed report…..
- Mid Stage Products (Phase II)
- Branaplam: Novartis
- Drug profiles in the detailed report…..
- Early Stage Products (Phase I/II)
- ACTX 401: Alcyone Therapeutics
- Drug profiles in the detailed report….
- Inactive Products
- Spinal muscular atrophy- Unmet Needs
- Spinal muscular atrophy- Market Drivers and Barriers
- Appendix
For further information on the Spinal Muscular Atrophy Pipeline therapeutics, reach out to Spinal Muscular Atrophy Unmet Needs and Analyst Views
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