Charcot-Marie-Tooth Disease Market
Nov 03, 2026
INOVIO’s VOCERYQ Receives FDA Accelerated Approval for Adult RRP; AbbVie Submits Etentamig Biologics License Application to FDA for R/R Multiple Myeloma; Setara Biosciences Raises $90 Million Series A to Advance Rare Disease Precision Medicines; BioMarin Expands Fabry Disease Portfolio With Exclusive Global License for Next-Generation ERT; Pfizer’s ELREXFIO Advances Multiple Myeloma Treatment With Positive Phase 3 Results
INOVIO Announces Accelerated FDA Approval for VOCERYQ in Adult RRP INOVIO announced that the U.S. Food and Drug Administration (FDA) has granted accelerated approval to VOCERYQ (doruxapapogene ralaplasmid and lalikinogene sifuplasmid-gxfd), formerly known as INO-3107, for reducing the frequency of surgical inter...
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Jul 10, 2026
The Race to Transform Charcot-Marie-Tooth Disease Treatment: Which Therapy Will Cross the Finish Line First?
Summary Charcot-Marie-Tooth disease is genetically heterogeneous, with more than 100 genes associated with different CMT disease subtypes. Currently, no approved therapies are available for the treatment of CMT disease, leaving patients with limited treatment options. As a result, there remains a significant...
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Mar 18, 2025
FDA Expands SOLIRIS for Pediatric Myasthenia Gravis; vTv’s Cadisegliatin Program Resumes as FDA Lifts Hold; ENCell’s EN001 Wins Orphan Drug Designation for Charcot-Marie-Tooth; FDA Accepts Sydnexis’ NDA for SYD-101 in Pediatric Myopia; Cambium Bio’s Potency Assay Strategy Cleared for Elate Ocular Phase III Trials
MDA Applauds FDA’s Expanded Approval of Soliris for Pediatric Generalized Myasthenia Gravis The Muscular Dystrophy Association (MDA) celebrates the FDA approval of an expanded indication for Alexion/AstraZeneca’s eculizumab (Soliris), now authorized for pediatric patients aged six and older with generalized myas...
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Jan 07, 2025
Chimerix Submits Dordaviprone NDA for Accelerated Approval in Recurrent H3 K27M-Mutant Diffuse Glioma to FDA; NMD Pharma’s NMD670 Receives FDA Orphan Drug Designation for Charcot-Marie-Tooth Disease; Verastem’s Avutometinib NDA Accepted for KRAS Mutant Ovarian Cancer; Capricor Submits BLA for Deramiocel in Duchenne Muscular Dystrophy; Axsome Completes Phase III Trial of AXS-05 for Alzheimer’s Agitation
Chimerix Submits Dordaviprone NDA for Accelerated Approval to FDA for Recurrent H3 K27M-Mutant Diffuse Glioma Chimerix announced the submission of a NDA to the FDA for dordaviprone. The application seeks accelerated approval for the treatment of recurrent H3 K27M-mutant diffuse glioma, a rare and aggressive brai...
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