Could you be 1 of the estimated 3300 patients in the US with the gene causing SORD Deficiency? You may have been diagnosed with CMT2, or distal hereditary motor neuropathy (dHMN). Join us as we learn more about SORD Deficiency and an investigational NEW Treatment with Shoshana Shendelman, Founder and CEO of Applied Therapeutics.
DTx Pharma Receives FDA Orphan Drug Designation for DTx-1252 for the Treatment of Charcot-Marie-Tooth Disease Type 1A (CMT1A)
Dtx today announced that the FDA has granted Orphan Drug Designation to DTx-1252, an investigational FALCON small interfering RNA (siRNA) therapeutic for the treatment of Charcot-Marie-Tooth Disease Type 1A (CMT1A).
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