DUBLIN--(BUSINESS WIRE)--The "Emerging Therapeutics for Rare Neuromuscular Diseases: Pipeline Analysis" report has been added to ResearchAndMarkets.com's offering. This research, "Emerging Therapeutics for Rare Neuromuscular Diseases: A Pipeline Analysis" explores emerging biologics across these six NMDs. 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.
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?
Factors Creating Pressure on Growth
Growth Opportunities Fuel the Growth Pipeline Engine
Predominant Emerging Biologic Classes Across NMDs
Sharp Growth in Investor Interest for Rare NMD Treatments Snapshot of Venture-backed Companies that Enable Biologics Progress
Patent Landscape - Biologics for NMDs
ALS: Biologics Pipeline Analysis
Biological Therapies for ALS ALS Oligonucleotide 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
Clinical Trial Progress in Biologics* for MG
DMD: Biologics Pipeline Analysis
Gene Therapies in the Emerging DMD Biologics Landscape Exon-Skipping 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 Biologics for DN Treatment
Biologics for Treatment Personalization in DN
Emerging Biologics for CIDP Treatment
Immunoglobulins for CIDP Treatments
Growth Opportunity 2: Cell and Gene Therapeutics
For more information about this report visit https://www.researchandmarkets.com/r/sgh4df