Spinal Muscular Atrophy Clinical Trials 2024 (Updated): EMA, PDMA, FDA Approval, Treatment Market, Medication, Companies, Drugs, MOA, ROA, and Recent Developments by DelveInsight

(Albany, USA) Spinal Muscular Atrophy pipeline constitutes 18+ key companies continuously working towards developing 20+ Spinal Muscular Atrophy treatment therapies, analysis of Clinical Trials, Therapies, Mechanism of Action, Route of Administration, and Developments analyzes DelveInsight.

"Spinal Muscular Atrophy Pipeline Insight, 2024″ report by DelveInsight outlines comprehensive insights into the present clinical development scenario and growth prospects across the Spinal Muscular Atrophy Market.

The Spinal Muscular Atrophy Pipeline report embraces in-depth commercial and clinical assessment of the pipeline products from the pre-clinical developmental phase to the marketed phase. The report also covers a detailed description of the drug, including the mechanism of action of the drug, clinical studies, NDA approvals (if any), and product development activities comprising the technology, collaborations, mergers acquisition, funding, designations, and other product-related details.

To know more in detail about Spinal Muscular Atrophy pipeline report offerings, click here: https://www.delveinsight.com/report-store/spinal-muscular-atrophy-sma-pipeline-insight?utm_source=openpr&utm_medium=pressrelease&utm_campaign=apr

Some of the key takeaways from the Spinal Muscular Atrophy Pipeline Report:
• Spinal Muscular Atrophy Companies across the globe are diligently working toward developing novel Spinal Muscular Atrophy treatment therapies with a considerable amount of success over the years.
• Leading Spinal Muscular Atrophy companies working in the market are 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, are developing therapies for the Spinal Muscular Atrophy treatment
• Spinal Muscular Atrophy emerging therapies such as - ACTX-401, Apitegromab, GYM329, and others are expected to have a significant impact on the Spinal Muscular Atrophy market in the coming years.
• On April 2024, Hoffmann-La Roche announced a Phase IV Open-Label Study Evaluating the Effectiveness and Safety of Risdiplam Administered as an Early Intervention in Pediatric Patients With Spinal Muscular Atrophy After Gene Therapy
• On April 2024, Genentech announced results of 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.
• On April 2024, Biogen announced results of a study whose primary objective is to examine the clinical efficacy of nusinersen administered intrathecally at higher doses to participants with spinal muscular atrophy (SMA), as measured by change in Children's Hospital of Philadelphia-Infant Test of Neuromuscular Disorders (CHOP-INTEND) total score (Part B); to examine the safety and tolerability of nusinersen administered intrathecally at higher doses to participants with SMA (Parts A and C).
• On January 2024, Novartis announced results of a Phase IIIb, Open-label, Single-arm, Multi-center Study to Evaluate the Safety, Tolerability and Efficacy of OAV101 Administered Intrathecally (1.2 x 10^14 Vector Genomes) to Participants 2 to < 18 Years of Age With Spinal Muscular Atrophy (SMA) Who Have Discontinued Treatment With Nusinersen (Spinraza®) or Risdiplam (Evrysdi®).
• On January 2024, NMD Pharma A/S announced results of a Phase 2, Randomised, Double-blind, Placebo-controlled, 2-way Crossover Study to Evaluate the Efficacy, Safety, and Tolerability of NMD670 in Ambulatory Adults With Type 3 Spinal Muscular Atrophy
• On December 2023, Novartis announced results of a Long-term Safety Study in Brazilian Patients With a Confirmed Diagnosis of Spinal Muscular Atrophy (SMA) Treated With Onasemnogene Abeparvovec (Zolgensma®) - ARISER Study.
• On May 2023, Biogen announced results of a study whose primary objective is to describe the natural history and utilization of disease modifying therapy (DMT) among adult Chinese participants with SMA linked to chromosome 5q (5q-SMA)

To know in detail about the Spinal Muscular Atrophy clinical trials and recent FDA approvals for Spinal Muscular Atrophy drugs, click here: https://www.delveinsight.com/sample-request/spinal-muscular-atrophy-sma-pipeline-insight?utm_source=openpr&utm_medium=pressrelease&utm_campaign=apr

Spinal Muscular Atrophy Overview
Spinal Muscular Atrophy (SMA) is a group of inherited disorders characterized by a loss of certain nerve cells in the spinal cord called motor neurons or anterior horn cells. Motor neurons receive the nerve impulses transmitted from the brain to the spinal cord (brainstem) and, in turn, transmit the impulses to the muscle via the peripheral nerves.

Spinal Muscular Atrophy Pipeline Therapeutics Assessment
• Spinal Muscular Atrophy Assessment by Product Type
• Spinal Muscular Atrophy By Stage and Product Type
• Spinal Muscular Atrophy Assessment by Route of Administration
• Spinal Muscular Atrophy By Stage and Route of Administration
• Spinal Muscular Atrophy Assessment by Molecule Type
• Spinal Muscular Atrophy by Stage and Molecule Type

DelveInsight's Spinal Muscular Atrophy Pipeline Report covers around 20+ products under different phases of clinical development like
• Late-stage products (Phase III)
• Mid-stage products (Phase II)
• Early-stage product (Phase I)
• Pre-clinical and Discovery stage candidates
• Discontinued & Inactive candidates
• Route of Administration

Emerging Spinal Muscular Atrophy Drugs Under Different Phases of Clinical Development Include:
• ACTX-401: Novartis
• Apitegromab: Scholar Rock
• GYM329: Roche
• And Many Others

Get a Free Sample PDF Report to know more about Spinal Muscular Atrophy Pipeline Assessment- https://www.delveinsight.com/report-store/spinal-muscular-atrophy-sma-pipeline-insight?utm_source=openpr&utm_medium=pressrelease&utm_campaign=apr

Spinal Muscular Atrophy Pipeline Analysis:
The Spinal Muscular Atrophy pipeline report provides insights into
• The report provides detailed insights about companies that are developing therapies for the Spinal Muscular Atrophy treatment with aggregate therapies developed by each company for the same.
• It accesses the Different therapeutic candidates segmented into early-stage, mid-stage, and late-stage of development for Spinal Muscular Atrophy Treatment.
• Spinal Muscular Atrophy key companies are involved in targeted therapeutics development with respective active and inactive (dormant or discontinued) projects.
• Spinal Muscular Atrophy Drugs under development based on the stage of development, route of administration, target receptor, monotherapy or combination therapy, a different mechanism of action, and molecular type.
• Detailed analysis of collaborations (company-company collaborations and company-academia collaborations), licensing agreement and financing details for future advancement of the Spinal Muscular Atrophy market.
The report is built using data and information traced from the researcher's proprietary databases, company/university websites, clinical trial registries, conferences, SEC filings, investor presentations, and featured press releases from company/university websites and industry-specific third-party sources, etc.

Further Spinal Muscular Atrophy product details are provided in the report. Download the Spinal Muscular Atrophy pipeline report to learn more about the emerging Spinal Muscular Atrophy therapies@ https://www.delveinsight.com/sample-request/spinal-muscular-atrophy-sma-pipeline-insight?utm_source=openpr&utm_medium=pressrelease&utm_campaign=apr

Scope of Spinal Muscular Atrophy Pipeline Drug Insight
• Coverage: Global
• Key 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
• Key Spinal Muscular Atrophy Therapies: AS-202, HK001, Tegoprubart, AP-101, BLZ945, ANX005, MN-166, TW001, BIIB067, and others
• Spinal Muscular Atrophy Therapeutic Assessment: Spinal Muscular Atrophy current marketed and Spinal Muscular Atrophy emerging therapies
• Spinal Muscular Atrophy Market Dynamics: Spinal Muscular Atrophy market drivers and Spinal Muscular Atrophy market barriers

Request for Sample Report @ https://www.delveinsight.com/sample-request/spinal-muscular-atrophy-sma-pipeline-insight?utm_source=openpr&utm_medium=pressrelease&utm_campaign=apr

Table of Contents
1. Spinal Muscular Atrophy Report Introduction
2. Spinal Muscular Atrophy Executive Summary
3. Spinal Muscular Atrophy Overview
4. Spinal Muscular Atrophy- Analytical Perspective In-depth Commercial Assessment
5. Spinal Muscular Atrophy Pipeline Therapeutics
6. Spinal Muscular Atrophy Late Stage Products (Phase II/III)
7. Spinal Muscular Atrophy Mid Stage Products (Phase II)
8. Spinal Muscular Atrophy Early Stage Products (Phase I)
9. Spinal Muscular Atrophy Preclinical Stage Products
10. Spinal Muscular Atrophy Therapeutics Assessment
11. Spinal Muscular Atrophy Inactive Products
12. Company-University Collaborations (Licensing/Partnering) Analysis
13. Spinal Muscular Atrophy Key Companies
14. Spinal Muscular Atrophy Key Products
15. Spinal Muscular Atrophy Unmet Needs
16. Spinal Muscular Atrophy Market Drivers and Barriers
17. Spinal Muscular Atrophy Future Perspectives and Conclusion
18. Spinal Muscular Atrophy Analyst Views
19. Appendix
20. About DelveInsight

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