Genetic Disorders

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Notizia - Recent Pharma, Healthcare and Biotech Happenings

Aminex Therapeutics Secures FDA Orphan Drug Designation for AMXT 1501 + DFMO in Neuroblastoma; Eccogene Gets FDA Nod to Begin Phase IIa MOSAIC Trial of ECC4703 + ECC0509 in MASH; Orca Bio’s Orca-T Earns FDA Priority Review for Hematologic Malignancies; Alto Neuroscience Gains FDA Fast Track for ALTO-101 in Cognitive Impairment Linked to Schizophrenia; Taysha Gene Therapies Wins FDA Breakthrough Therapy Status for TSHA-102 in Rett Syndrome

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Merck Wins FDA Approval for KEYTRUDA QLEX for Subcutaneous Use in Adults With Solid Tumors; Incyte Gains FDA Nod for OPZELURA Cream in Children Aged 2–11 With Atopic Dermatitis; Minovia Therapeutics Receives FDA Fast Track Designation for MNV-201 in Myelodysplastic Syndrome; MavriX Bio Secures FDA Fast Track for MVX-220 Gene Therapy in Angelman Syndrome; Biocon Biologics Gets FDA Approval for Denosumab Biosimilars BOSAYA and AUKELSO

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The Rise of AI-Powered Point-of-Care Diagnostics: Transforming Real-Time Patient Care

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NeuroNOS’ BA-101 Granted FDA Orphan Drug Status for Glioblastoma; Takeda’s VONVENDI Approval Expanded for Von Willebrand Disease in Adults and Children; Telix Gains FDA Nod on Resubmission Pathway for TLX101-CDx NDA; Saol Therapeutics Receives FDA CRL for SL1009 in Pyruvate Dehydrogenase Complex Deficiency; FDA Grants Breakthrough Therapy Designation to Boehringer’s HERNEXEOS for HER2-Mutant Advanced NSCLC

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Precigen’s PAPZIMEOS Wins Full FDA Approval for Recurrent Respiratory Papillomatosis; Tonix’s Tonmya Becomes First FDA-Approved Fibromyalgia Treatment in Over 15 Years; FDA Extends BLA Review for REGENXBIO’s RGX-121 in MPS II Patients; SystImmune’s Izalontamab Brengitecan (EGFRxHER3 ADC) Gets FDA Breakthrough Designation for EGFR-Mutated NSCLC; Soligenix Granted FDA Orphan Drug Status to SGX945 for Behçet’s Disease Therapy Following Phase II Success

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Anbogen’s ABT-301 Cleared by FDA for Phase I/II Colorectal Cancer Trial; Dyne’s DYNE-251 Gets FDA Breakthrough Tag for Duchenne Muscular Dystrophy; BMS’s BREYANZI Accepted for FDA Priority Review in MZL; Clarametyx’s CMTX-101 Earns FDA Fast Track and QIDP for Infections; Anixa Begins FDA-Approved IND Transfer for Phase II Breast Cancer Vaccine Trial

Feature-Interleukin-2-targeting Therapies Journey from Bench to Bedside

Understanding Immunohistochemistry: A Key Tool in Biomedical Research and Market Insights

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Sarepta Therapeutics Pauses ELEVIDYS Shipments in U.S. After FDA Intervention Over Patient Deaths; ImCheck’s ICT01 Granted FDA Orphan Drug Designation for Acute Myeloid Leukemia; RedHill Biopharma Gets Positive FDA Feedback on RHB-204 for Crohn’s Disease Approval Pathway; FDA Grants Fast Track Designation to ProMIS Neurosciences’ PMN310 for Alzheimer’s Disease; Oruka Therapeutics Receives IND Clearance for Phase IIa Trial of ORKA-001 in Psoriasis

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KalVista’s EKTERLY: First-Ever Oral Hereditary Angioedema Treatment

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Merck’s WINREVAIR Granted FDA Priority Review for Pulmonary Arterial Hypertension; KalVista’s EKTERLY Approved by FDA as First Oral On-Demand Therapy for Hereditary Angioedema; Fujifilm’s FF-10832 Receives FDA Orphan Drug Designation for Biliary Tract Cancer; Mustang Bio’s MB-101 Granted FDA Orphan Drug Designation for Glioblastoma and Astrocytomas; Denali’s Tividenofusp Alfa Accepted for FDA Priority Review for Hunter Syndrome

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Discover How Learning Disability Treatment Landscape is Evolving with the Emergence of Digital Assistant Technologies

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Sobi’s GAMIFANT Approved by FDA as First Treatment for Macrophage Activation Syndrome in Still’s Disease; GSK Cuts RTS,S Malaria Vaccine Price for Children in Endemic Regions to Under $5; Schrödinger’s SGR-1505 Receives FDA Fast Track Designation for Relapsed/Refractory Waldenström Macroglobulinemia; Ultragenyx’s GTX-102 Granted FDA Breakthrough Therapy Designation for Angelman Syndrome; Eli Lilly’s AMYVID Label Updated by FDA to Support Alzheimer’s Disease Diagnosis

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Celltrion Announces FDA Nod for New STEQEYMA Presentation to Broaden Pediatric Use; CSL’s ANDEMBRY Gets FDA Nod as First Factor XIIa Inhibitor for Hereditary Angioedema; Ocugen’s OCU410ST Cleared by FDA to Begin Phase II/III Trial in Stargardt Disease; Milestone Pharmaceuticals Submits FDA Response for CARDAMYST in PSVT; AbbVie’s MAVYRET Approved by FDA as First and Only Therapy for Acute Hepatitis C

thymidine-kinase-2-deficiency-treatment-landscape

Thymidine Kinase 2 Deficiency Treatment: Advances in Therapeutic Strategies and Clinical Development

Types of Mitochondrial Disorders and the Science Behind Them: A Journey from Dysfunction to Discovery

Types of Mitochondrial Diseases and the Science Behind Them: A Journey from Dysfunction to Discovery

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Beam’s BEAM-302 Earns FDA Orphan Drug Tag for AATD; Incannex’s IHL-42X Moves to Phase III After FDA Protocol OK; Kura and Kyowa Kirin’s Ziftomenib Gets FDA Priority Review in NPM1-Mutant AML; GSK’s Linerixibat NDA Accepted by FDA for PBC-Linked Pruritus; Ascendis’ TransCon CNP Granted FDA Priority Review for Achondroplasia

niemann-pick-disease-types-therapies-overview

Everything You Need to Know About Niemann-Pick Disease – From Types to Therapies

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