Myotonic Dystrophy
Oct 28, 2025
Syndax Pharmaceuticals Receives FDA Approval for REVUFORJ; FDA Approves BLENREP for Adults with Relapsed/Refractory Multiple Myeloma; Novartis to Acquire Avidity for $12 Billion; BridgeBio Reports Positive Phase 3 Results for Small Molecule BBP-418 in LGMD2I/R9 FORTIFY Study; Eli Lilly to Acquire Adverum Biotechnologies
Syndax Pharmaceuticals Receives Approval for REVUFORJ from the FDA Syndax Pharmaceuticals announced that the FDA has approved REVUFORJ (revumenib) for the treatment of relapsed or refractory acute myeloid leukemia with a susceptible nucleophosmin 1 (NPM1) mutation in adult and pediatric patients one year and old...
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Jan 28, 2025
FDA Approves LEQEMBI IV Dosing for Early Alzheimer’s; Vanda Accepts FDA Hearing on Tradipitant for Gastroparesis; Cartesian Gains FDA Protocol Approval for Myasthenia Gravis Trial; Zai Lab Secures FDA Orphan Status for DLL3 ADC in SCLC; Dyne Receives FDA Fast Track for DYNE-101 in Myotonic Dystrophy
FDA Approves LEQEMBI® (Lecanemab-Irmb) IV Maintenance Dosing for Early Alzheimer’s Disease Treatment Eisai Co., Ltd. and Biogen Inc. announced that the FDA has approved the Supplemental Biologics License Application (sBLA) for LEQEMBI (lecanemab-irmb), allowing intravenous (IV) maintenance dosing every four week...
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Jan 30, 2024
Merck’s KEYTRUDA as Adjuvant Therapy for RCC Patients; BMS Receives Positive CHMP Opinion for CAR T Cell Therapy Abecma for Multiple Myeloma; FDA Approves Dupixent for Eosinophilic Esophagitis; Juvena Receives FDA Orphan Drug Designation for JUV-161; European Commission Authorizes GSK’s Omjjara; ENHERTU Granted Priority Review in the US for for metastatic HER2-positive Solid Tumors
Merck’s KEYTRUDA Reduced the Risk of Death by 38% Versus Placebo as Adjuvant Therapy for Patients With Renal Cell Cancer (RCC) at an Increased Risk of Recurrence Following Nephrectomy Merck, also known as MSD beyond the United States and Canada, has revealed findings from the Phase III KEYNOTE-564 trial, which a...
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Dec 12, 2022
Myotonic Dystrophy Treatment: In Search of Effective Weaponry to Kick Out Toxic RNAs Weakening Muscles
Myotonic dystrophy (DM), known as "the most variable of all diseases found in medicine," is a multi-systemic genetic disorder that is the most common form of adult-onset muscular dystrophy. myotonic dystrophy is the only type of muscular dystrophy that affects cognition and brain function, in addition to the heart,...
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