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May 05, 2026
The United Arab Emirates has unveiled two gene therapy projects targeting autism, epilepsy, and rare brain disorders in children. The initiatives focus on underlying genetic causes and earlier diagnosis.
Apr 21, 2026
Xenon Pharmaceuticals reported positive Phase III X-TOLE2 results for azetukalner in focal onset seizures. The company plans an FDA new drug application in Q3 2026.
Apr 03, 2026
The FDA has approved nivolumab with chemotherapy for pediatric and adult Hodgkin lymphoma patients aged 12+, based on clinical trial data showing improved survival. Separately, the agency cleared investigational new drug applications for FG001 for brain cancer surgery visualization and FRF-001 gene therapy for FOXG1 syndrome, allowing both to proceed to clinical trials.
Mar 31, 2026
The FDA has accepted Praxis Precision Medicines' NDA for relutrigine with priority review and set a PDUFA target date of September 27, 2026. The therapy targets SCN2A and SCN8A developmental and epileptic encephalopathies and could become the first approved treatment for these rare conditions. The company has bolstered its cash position to support commercial launch preparations.
Mar 23, 2026
The global generic drugs market reached $389 billion in 2024 and is projected to grow to $675 billion by 2033. In the U.S., generics account for 91% of prescriptions but only 18% of drug spending, saving over $373 billion annually. Biosimilar adoption remains below 20% despite some products reaching over 60% market share within three years.
Mar 14, 2026
New research reveals how ketamine rewires brain receptors to rapidly relieve severe depression, while separate trials demonstrate success with personalized brain stimulation and targeted drug therapy for treatment-resistant cases.
Mar 14, 2026
The FDA has authorized the first human trial of an allogeneic stem cell therapy for epilepsy, developed by Shanghai-based Unixell Biotechnology. The therapy uses donor-derived stem cells to produce GABA and suppress seizures in drug-resistant patients.
Mar 09, 2026
Xenon Pharmaceuticals will announce topline data from its Phase 3 X-TOLE2 study of azetukalner in patients with focal onset seizures on March 9, 2026, during a conference call at 8:00 a.m. ET.
Mar 05, 2026
New treatments for rare pediatric neurological conditions show promise, with zorevunersen reducing seizures in Dravet syndrome patients and an SMA therapy under FDA review following stem cell research breakthroughs.
Mar 04, 2026
Roche signed an agreement to invest $478 million in Korea clinical trials over five years, marking the largest foreign pharmaceutical investment. Korean companies are also securing pre-approval licensing deals globally, demonstrating growing competitiveness.
Mar 03, 2026
University of Melbourne receives $2.1 million for quantum-enabled brain-on-chip platform for neurological diseases, while Evogene partners with Queensland University of Technology on AI-driven cancer therapeutics targeting chemotherapy resistance.
Feb 24, 2026
Multiple analysts have raised price targets on Praxis Precision Medicines, with Wolfe Research initiating coverage at $500 and Guggenheim increasing its target to $800, citing strong market potential for ulixacaltamide in essential tremor.
Feb 16, 2026
The U.S. Food and Drug Administration has approved an Investigational New Drug application for FRF-001, a viral gene therapy for FOXG1 syndrome developed at the University at Buffalo. The first-in-human trial will be independently sponsored by the FOXG1 Research Foundation.
Feb 19, 2026
Praxis Precision Medicines submitted NDAs for ulixacaltamide and relutrigine to the FDA, reported cash and investments of $926 million as of December 31, 2025, and disclosed a net loss of $303.3 million for full-year 2025.
Feb 16, 2026
Researchers at Monash University have identified DNA sequence variations in mothers that modify the risk of birth defects from valproic acid, an anti-seizure medication, paving the way for a genetic test to identify who can safely take the drug during pregnancy.
Dec 30, 2025
Praxis Precision Medicines has submitted new drug applications for ulixacaltamide in essential tremor and relutrigine in rare epilepsies, both granted Breakthrough Therapy Designation. The company reported $926 million in cash as of December 31, 2025.
Aug 15, 2025
Drug Hunter's monthly molecule selections from July through December 2025 feature innovative small molecule candidates targeting pain, epilepsy, cancer, infectious diseases, and metabolic disorders, including non-opioid pain agents and precision therapies for rare conditions.
