Disease or Syndrome – Page 105

FDA Approves Vadadustat (Vafseo) for Anemia in Chronic Kidney Disease Patients on Dialysis and Daprodustat (Jesduvroq) for Dialysis-Dependent CKD Adults

1 year ago talkbio0Tagged Anemia, Approved, Black Box Warning, Chronic Kidney Diseases, Daprodustat, Effectiveness, Hemoglobin concentration result, Jesduvroq, Oral Medication, post-marketing safety, Safety, United States Food and Drug Administration, vadadustat, Vafseo

Anemia, Chronic Kidney Disease (CKD), Vadadustat (Vafseo), Daprodustat (Jesduvroq), Oral medication, FDA approval, Hemoglobin levels, Black Box Warning, Efficacy and safety data, Post-marketing safety data

Pfizer Discontinues Phase III Trial for Sickle Cell Treatment Amid Challenges in Patient Recruitment

1 year ago talkbio0Tagged Anemia, Sickle Cell, ClinicalTrials.gov, Grail, inclacumab, Pfizer, Phase 3 Clinical Trials, Poor Accrual, Recruitment Challenges, THRIVE Program

Pfizer, Sickle Cell Disease, Phase III Study, Inclacumab, Poor Accrual, Recruitment Challenges, THRIVE Program, ClinicalTrials.gov, Gene Therapies

Praxis’ Mid-Stage Triumph Propels Advancement of Novel Epilepsy Drug

1 year ago talkbio0Tagged Adult Focal Onset, concept, Efficacy Study, Epilepsy, Functionally Selective Small Molecule,, Hyperexcitable State, Phase IIa, PRAX-628, Praxis Precision Medicines, Sodium Channel, Success

Praxis Precision Medicines, PRAX-628, Functionally Selective Small Molecule, Hyperexcitable State of Sodium Channels, Adult Focal Onset Epilepsy Treatment, Phase IIa Proof of Concept Study Success, Second Half of 2024 Efficacy Study Planned

Groundbreaking Data Revealed for Genetic Epilepsy Drug STK-001 by Stoke Therapeutics, Exhilarating Investors

1 year ago talkbio0Tagged Antisense Oligonucleotides, Genetic Epilepsy Drug, Infantile Severe Myoclonic Epilepsy, investor, Level of interest, Positive Clinical Trial, Seizures, STK-001, Stoke

Stoke Therapeutics, STK-001, Genetic Epilepsy Drug, Dravet Syndrome, Antisense Oligonucleotide, Seizure Reduction, Positive Clinical Trial Data, Investor Enthusiasm

AstraZeneca’s Ultomiris Gains FDA Approval for Rare Autoimmune Condition – NMOSD

1 year ago talkbio0Tagged Approved, AstraZeneca, Autoimmune Diseases, dosing, Infrequent, Long-acting C5 complement inhibitor, Neuromyelitis Optica, Rating (action), ravulizumab-cwvz, Reduced, Relapse, Ultomiris, United States Food and Drug Administration, With frequency

AstraZeneca, Ultomiris, ravulizumab-cwvz, FDA Approval, Rare Autoimmune Disease, Neuromyelitis Optica Spectrum Disorder (NMOSD), Long-acting C5 complement inhibitor, Phase 3 CHAMPION-NMOSD Trial, Reduced Relapse Rate, Dosing Frequency

Axsome Therapeutics’ Small Molecule AXS-12 Successful in Phase III Narcolepsy Trial

1 year ago talkbio0Tagged AXS-12, Axsome, Cataplexy, Drowsiness, Improved, memory/, Narcolepsy, Phase 3 Clinical Trials, reboxetine, safety profile, Severities

Axsome Therapeutics, AXS-12 (reboxetine), Phase III trial, Narcolepsy, Cataplexy reduction, Improved cognition/ memory/ sleepiness severity, Safety profile

Alexion Expands Indications for Long-Acting C5 Complement Inhibitor Ultomiris in Rare Autoimmune Disorder NMOSD

1 year ago talkbio0Tagged Alexion, Annals, Approved, AstraZeneca Rare Disease, C5 complement inhibitor, CHAMPIER-NMOSD trial, generalised myasthenia gravis, gMG, long-acting, Marc Dunoyer, Neurology speciality, Neuromyelitis Optica, Paroxysmal nocturnal hemoglobinuria, Publications, ravulizumab-cwvz, Ultomiris, United States Food and Drug Administration

Ultomiris, ravulizumab-cwvz, C5 complement inhibitor, neuromyelitis optica spectrum disorder (NMOSD), FDA approval, long-acting, zero relapses, CHAMPIER-NMOSD trial, Annals of Neurology publication, Marc Dunoyer, AstraZeneca Rare Disease, Alexion, generalised myasthenia gravis (gMG), paroxysmal nocturnal haemoglobinuria (PNH).

FDA Grants Emergency Authorization for Invivyd’s Pemgarda Antibody Prophylactic Against COVID-19 in Immunocompromised Individuals

1 year ago talkbio0Tagged Adolescent (age group), Adults/, Approved, Condoms, Male, COVID19 (disease), Emergency Use Authorization, Half-Life Extended Monoclonal Antibody, immune compromise, Immunocompromised Host, Invivyd, Moderate (severity modifier), Pemgarda, pemivibart, Pre-Exposure Prophylaxis, Prophylactic treatment, Severe (severity modifier), United States Food and Drug Administration, VYD222

Invivyd, Pemgarda (pemivibart; formerly VYD222), Half-Life Extended Monoclonal Antibody, COVID-19 Prophylactic, Immunocompromised Patients, Emergency Use Authorization (EUA), FDA Approval, Moderate to Severe Immune Compromise, Pre-exposure Prophylaxis (Prevention) of COVID-19 in Adults/ Adolescents