NEW YORK, Dec. 1, 2022 /PRNewswire/ --
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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.Biologics presents a personalized and targeted method to treat NMDs and is a promising treatment class.
These indications are likely to witness new drug developments.
Frost & Sullivan’s research, “Emerging Therapeutics for Rare Neuromuscular Diseases: A Pipeline Analysis†explores emerging biologics across these six NMDs.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. 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.Key Points DiscussedWhat 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?
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