Organism Attribute

FDA Rejection Derails PTC Therapeutics’ Rare Disease Hopeful

3 weeks ago talkbio0Tagged Clinical Trials, concerns, Effectiveness, factor IX, Failure (biologic function), Friedreich 's ataxia, Rare Diseases, Rejection, Response process, United States Food and Drug Administration, vatiquinone

PTC Therapeutics; FDA rejection; vatiquinone; Friedreich’s ataxia; rare disease; efficacy concerns; complete response letter; clinical trial failure

Response Pharma: RDX-002 Limits Weight Regain After GLP-1 Therapy – Positive Phase II Results Announced

4 weeks ago talkbio0Tagged cardiometabolic health, Discontinuation (procedure), Glucagon-Like Peptide 1, iMTP, Inhibitor, Obesity, Phase 2 Clinical Trials, postprandial, RDX-002, Response process, Triglycerides, weight regain

Response Pharmaceuticals; RDX-002; GLP-1 discontinuation; weight regain; obesity; Phase II clinical trial; postprandial triglycerides; cardiometabolic health; iMTP inhibitor

Spine BioPharma’s Phase 3 Back Pain Drug Fails to Meet Primary Endpoint, Attributes Miss to High Sham Response

1 month ago talkbio0Tagged Chronic low back pain, Clinical Trials, Intervertebral Disc Degeneration, primary endpoint, Response process, salicylhydroxamic acid, SB-01, Spine BioPharma

Spine BioPharma; Phase 3 trial; chronic low back pain; degenerative disc disease; SB-01; primary endpoint; sham response; clinical trial results

FDA Rejects Capricor’s Cell Therapy for Duchenne Muscular Dystrophy

2 months ago talkbio0Tagged Capricor Therapeutics, Cardiomyopathies, Cell Therapy, Clinical Data, Complete, deramiocel, HOPE-3 trial, Muscular Dystrophy, Duchenne, Phase 3, Rejection, Response process, United States Food and Drug Administration

FDA rejection; Capricor Therapeutics; deramiocel; Duchenne muscular dystrophy; DMD; cell therapy; cardiomyopathy; clinical data; Phase 3 HOPE-3 trial; Complete Response Letter

Federal Judge Rules NIH Grant Terminations Illegal, Orders Restoration of Hundreds of Grants

3 months ago talkbio0Tagged Biomedical Research, DEI, federal judge, Gender, health disparity, LGBTQ+, NIH Mouse, public health medicine (field), Racism, Sense of identity (observable entity), terminations, Trump administration

NIH; grant terminations; federal judge; racial discrimination; DEI; gender identity; Trump administration; biomedical research; public health; LGBTQ+; health disparities

Analysts Downplay Intellia Adverse Event, but Gene Therapy Safety Remains Under Scrutiny

3 months ago talkbio0Tagged Adverse event, ATTR-CM, Event, Gene Modification, gene therapy, Intellia, liver transaminase, MAGNITUDE trial, Response process, Safety, Stock Decline

Intellia Therapeutics; gene therapy; safety event; adverse event; MAGNITUDE trial; liver transaminase; stock decline; ATTR-CM; gene editing; analyst response

France pauses chikungunya vaccinations in the elderly after patient death

4 months ago talkbio0Tagged Adverse event, Age, Chikungunya Fever, Disease Outbreaks, Elderly (population group), France, Latex Fixation Tests, older, Reunion Island, Vaccination, Vaccines

Vaccines, Chikungunya Fever, Elderly (population group), Adverse event, France, Latex Fixation Tests, Reunion Island, Disease Outbreaks, Age, older, Vaccination

CDC advisors broaden RSV vaccine recommendations to at-risk adults in their 50s

5 months ago talkbio0Tagged 50–59, Adult, Age, Arexvy, broaden, Centers for Disease Control and Prevention (U.S.), Chronic Condition, Recommendation, Respiratory syncytial virus, Severe (severity modifier), Vaccines

Adult, Respiratory syncytial virus, Centers for Disease Control and Prevention (U.S.), Vaccines, Recommendation, Age, Arexvy, Chronic Condition, Severe (severity modifier), 50–59, broaden

Alzheon Investigates Subgroup Data Following Phase 3 Trial of ALZ-801 in Alzheimer’s Disease

5 months ago talkbio0Tagged ALZ-801, Alzheimer 's disease, Amyloid-targeting therapy, Apolipoprotein E4, Genotype, Mental deterioration, Mild cognitive disorder, Phase 3 trial, Precision Medicine, Subgroup A Nepoviruses, Valiltramiprosate

Alzheimer’s disease , ALZ-801 , Valiltramiprosate , APOE4 genotype , Mild cognitive impairment (MCI) , Amyloid-targeting therapy , Phase 3 trial results , Cognitive decline , Subgroup analysis , Precision medicine

FDA Rejects Milestone Pharmaceuticals’ Etripamil Nasal Spray for PSVT

6 months ago talkbio0Tagged Cardamyst, Complete, etripamil, Milestone, Paroxysmal supraventricular tachycardia, Rejection, Response process, United States Food and Drug Administration

Etripamil, Cardamyst, PSVT, FDA rejection, Complete Response Letter, manufacturing issues, Milestone Pharmaceuticals