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Ocugen Announces Positive EMA Scientific Advice for OCU410ST Gene Therapy for Stargardt Disease

Ocugen, Inc. announced positive scientific advice from the European Medicines Agency (EMA) regarding the approval pathway for OCU410ST, a modifier gen...
Other Clinical Trial Updates

Amgen's Repatha Phase 3 VESALIUS-CV Trial Data to be Presented at AHA 2025

Amgen announced that new data from its cardiovascular portfolio, including results from the Phase 3 VESALIUS-CV clinical trial of Repatha (evolocumab)...

Genentech's Gazyva Shows Positive Phase III Data with Significant Reduction in Disease Activity for Systemic Lupus Erythematosus

Genentech announced positive results from the Phase III ALLEGORY study of Gazyva in adults with systemic lupus erythematosus (SLE) on standard therapy...

GeneSight Test Shows Faster Remission and Response in Depression Treatment: Post-Hoc Analysis

Myriad Genetics announced a post-hoc analysis of the PRIME study, revealing that patients with Major Depressive Disorder (MDD) treated based on GeneSi...

Foghorn Therapeutics Announces Updates for Selective ARID1B, CBP, and EP300 Degrader Programs

Foghorn Therapeutics announced updates on its Selective ARID1B, CBP, and EP300 degrader programs. The Selective ARID1B degrader selectively binds and ...

Wave Life Sciences Announces Positive Target Engagement Data from INLIGHT Clinical Trial of WVE-007 for Obesity

Wave Life Sciences announced positive Activin E target engagement data from the INLIGHT clinical trial of WVE-007, a GalNAc-siRNA, for obesity. The tr...

Anaptys Announces Positive Phase 2b Results for Rosnilimab in Rheumatoid Arthritis

AnaptysBio announced positive late-breaking data from its Phase 2b trial of rosnilimab in rheumatoid arthritis (RA). The study demonstrated deepening ...

AbbVie Announces Positive Phase 3 Results for Upadacitinib (RINVOQ®) in Vitiligo

AbbVie announced positive topline results from two replicate Phase 3 studies evaluating upadacitinib (RINVOQ®) in adult and adolescent patients with n...

TG Therapeutics Completes Enrollment in Phase 3 ENHANCE Trial of Simplified Dosing for BRIUMVI in Multiple Sclerosis

TG Therapeutics announced the completion of enrollment in the Phase 3 ENHANCE trial, which is evaluating a simplified dosing schedule for BRIUMVI (ubl...

Kyverna Therapeutics Announces Positive Phase 2 Interim Data for KYV-101 in Generalized Myasthenia Gravis

Kyverna Therapeutics announced positive interim Phase 2 data from the KYSA-6 study of KYV-101 in generalized myasthenia gravis (gMG). The data showed ...

Sionna Therapeutics Announces Phase 1 Data for SION-719 and SION-451 and Preclinical Data on NBD1 Stabilizers in Cystic Fibrosis

Sionna Therapeutics announced the presentation of Phase 1 clinical trial data for SION-719 and SION-451, first-in-class NBD1 stabilizers, at the 2025 ...

Daewoong Pharmaceutical's Bersiporocin Receives Positive IDMC Safety Review in Phase 2 IPF Trial

Daewoong Pharmaceutical announced that Bersiporocin (DWN12088), its first-in-class drug candidate for idiopathic pulmonary fibrosis (IPF), received a ...

Innovent's Mazdutide Demonstrates Superior Glycemic Control and Weight Loss Compared to Semaglutide in Phase 3 DREAMS-3 Trial

Innovent Biologics announces positive results from its Phase 3 DREAMS-3 clinical trial of mazdutide, a glucagon-like peptide-1 (GLP-1) and glucagon (G...

Xencor Presents Phase 1 Data for XmAb819 in Clear Cell Renal Cell Carcinoma

Xencor announced initial results from the Phase 1 dose-escalation study of XmAb819, a first-in-class ENPP3 x CD3 bispecific T-cell engager, in patient...

Cullinan Therapeutics Presents Preclinical Data for CLN-978 Supporting Clinical Development in Autoimmune Diseases at ACR Convergence 2025

Cullinan Therapeutics presented preclinical data for CLN-978, a CD19xCD3 bispecific T cell engager, at the American College of Rheumatology (ACR) Conv...

Lilly's Omvoh (mirikizumab-mrkz) Shows Improvement in Bowel Urgency for Ulcerative Colitis Patients

Eli Lilly and Company announced positive data from the Phase 3b LUCENT-URGE study, demonstrating that Omvoh (mirikizumab-mrkz) significantly improved ...

Moderna's Phase 3 CMV Vaccine Trial Fails to Meet Primary Efficacy Endpoint

Moderna announced that its Phase 3 trial evaluating mRNA-1647, an investigational cytomegalovirus (CMV) vaccine, did not meet its primary efficacy end...

Kyverna Therapeutics Announces Conference Call on Interim Phase 2 Data of KYV-101 in Generalized Myasthenia Gravis

Kyverna Therapeutics will host a conference call to discuss interim data from the KYSA-6 Phase 2 clinical trial evaluating KYV-101 in generalized myas...

Rozanolixizumab Prevents Congenital Heart Block Recurrence in High-Risk Pregnancy

NYU Langone Health researchers found that rozanolixizumab, a neonatal Fc receptor (FcRn) inhibitor, can prevent congenital heart block in newborns of ...

OS Therapies Announces Positive Survival Data for OST-HER2 in Metastatic Osteosarcoma Subgroups

OS Therapies Inc. announced additional overall and event free survival data from its Phase 2b clinical trial of OST-HER2 in recurrent, fully resected,...

AskBio Announces Positive 12-Month Data from Phase 1 Trial of AB-1002 Gene Therapy for Congestive Heart Failure in Nature Medicine

AskBio, a subsidiary of Bayer AG, announced the publication of 12-month data from its Phase 1 trial of AB-1002 gene therapy for congestive heart failu...

IDEAYA Biosciences Announces Positive Phase 2 Data for Darovasertib in Neoadjuvant Primary Uveal Melanoma

IDEAYA Biosciences announced positive Phase 2 clinical data for darovasertib in the neoadjuvant setting of primary uveal melanoma (UM). The OptimUM-09...

Sionna Therapeutics Initiates Phase 2a Trial of SION-719 for Cystic Fibrosis

Sionna Therapeutics has initiated the PreciSION CF Phase 2a trial to evaluate SION-719, a novel NBD1 stabilizer, in cystic fibrosis (CF) patients. The...

Apnimed Presents Phase 3 Data for AD109 in Obstructive Sleep Apnea at CHEST 2025

Apnimed announced additional data from its Phase 3 SynAIRgy and LunAIRo trials evaluating AD109 (aroxybutynin 2.5 mg/atomoxetine 75 mg) for obstructiv...

CatalYm Presents Positive Long-Term Phase 1/2a Data for Visugromab in CPI-Refractory Tumors at ESMO 2025

CatalYm announced updated long-term data from its GDFATHER-1/2a trial at ESMO Congress 2025, showcasing the potential of visugromab to reverse resista...

Olema Oncology Announces Positive Phase 1b/2 Data of Palazestrant Plus Ribociclib in ER+/HER2- Metastatic Breast Cancer at ESMO 2025

Olema Oncology announced updated data from the Phase 1b/2 study of palazestrant in combination with ribociclib in patients with ER+/HER2- advanced or ...

Disc Medicine to Present Phase 1b Data of DISC-0974 in CKD Patients with Anemia at ASN Kidney Week 2025

Disc Medicine will present data from its Phase 1b trial of DISC-0974 in patients with non-dialysis-dependent chronic kidney disease (NDD-CKD) and anem...

SystImmune and Bristol Myers Squibb Announce Phase I Results of Iza-bren (EGFR x HER3 ADC) in Advanced Solid Tumors

SystImmune and Bristol Myers Squibb announced the first global Phase I results of iza-bren (BL-B01D1), an EGFR x HER3 bispecific antibody-drug conjuga...

Exelixis Announces Positive Phase 3 Results for Zanzalintinib and Atezolizumab Combination in Metastatic Colorectal Cancer

Exelixis announced detailed results from the Phase 3 STELLAR-303 trial evaluating zanzalintinib in combination with atezolizumab versus regorafenib in...

AlphaMedix Phase 2 Data Shows Potential in Gastroenteropancreatic Neuroendocrine Tumors

AlphaMedix phase 2 study (ALPHAMEDIX-02) evaluating AlphaMedix (212Pb-DOTAMTATE) in patients with advanced gastroenteropancreatic neuroendocrine tumor...

AB Science Announces Initial Phase 1 Data for AB8939 and Venetoclax Combination in Refractory or Relapsed Acute Myeloid Leukemia

AB Science SA announced initial Phase 1 data for the combination of AB8939 with venetoclax for treating refractory or relapsed acute myeloid leukemia ...

Praxis Announces Positive Phase 3 Results for Ulixacaltamide in Essential Tremor

Praxis Precision Medicines announced positive topline results from two pivotal Phase 3 studies (Essential3 program) of ulixacaltamide HCl in patients ...

Vor Bio Announces Positive Phase 3 Results of Telitacicept in Systemic Lupus Erythematosus Published in NEJM

Vor Bio announces the publication of positive results from a Phase 3 trial in China evaluating telitacicept for systemic lupus erythematosus (SLE) in ...

Nuvectis Pharma Announces Upcoming Presentations for NXP900 at AACR-NCI-EORTC Conference

Nuvectis Pharma, Inc. announced upcoming poster presentations for NXP900 at the 2025 AACR-NCI-EORTC International Conference on Molecular Targets and ...

Aicuris' Pritelivir Meets Primary Endpoint in Phase 3 Trial for Refractory Herpes Simplex Virus in Immunocompromised Patients

Aicuris Anti-infective Cures AG announced positive results from its Phase 3 trial (PRIOH-1) of pritelivir for treating refractory Herpes Simplex Virus...

Spago Nanomedical Announces Positive Data and Dose Increase Recommendation for 177Lu-SN201 in Phase I/IIa Tumorad-01 Study

Spago Nanomedical announced that the Data Monitoring Committee (DMC) recommended a dose increase in the ongoing Phase I/IIa clinical trial Tumorad-01 ...

Gilead and Kite Present New Cancer Data at ESMO 2025, Highlighting Trodelvy's Potential in Metastatic TNBC

Gilead and Kite will present new data at the ESMO 2025 Congress, showcasing progress in cancer treatment. Key highlights include Phase 3 ASCENT-03 stu...

Boehringer and Click Therapeutics present positive Phase III data for CT-155 in Schizophrenia

Boehringer Ingelheim and Click Therapeutics announced positive results from the Phase III CONVOKE study of CT-155, an investigational prescription dig...

Encoded Therapeutics Announces Positive Preclinical Data for Non-Opioid Gene Therapy Candidate for Chronic Pain

Encoded Therapeutics announced preclinical data for its AAV9 microRNA (miRNA) gene therapy candidate targeting SCN9A (NaV1.7) for chronic pain. The da...

Taysha Gene Therapies Presents New Data on TSHA-102 for Rett Syndrome at CNS Annual Meeting

Taysha Gene Therapies announced new supplemental data analysis from Part A of the REVEAL Phase 1/2 trials evaluating TSHA-102 in females with Rett syn...

GlyTR Therapeutics Announces Publication of Pan-Cancer Immunotherapy Platform Study in Cell

GlyTR Therapeutics announced the publication of a study in Cell detailing their Glycan-Dependent T cell Recruiter (GlyTR1 & GlyTR2) therapeutics. Thes...

BrYet US Receives Approval to Initiate Phase 1/2 Trial of ML-016 in Advanced Cancers with Lung and/or Liver Involvement in Australia

BrYet US, Inc. has received approval to initiate a first-in-human Phase 1/2 clinical trial of ML-016 in Australia. The trial will evaluate the safety,...

Prilenia and Ferrer Announce Phase 3 ALS Study of Pridopidine at NEALS 2025

Prilenia Therapeutics and Ferrer announced the presentation of their planned pivotal Phase 3 study of pridopidine in Amyotrophic Lateral Sclerosis (AL...

Editas Medicine Announces EDIT-401 Oral Presentation at ESGCT Congress and Participation in Investor Conferences

Editas Medicine announced an oral presentation on EDIT-401, a CRISPR gene editing medicine for lowering LDL-cholesterol by upregulating LDLR, at the E...

Myrtelle Announces Encouraging Interim Results from Phase 1/2 Gene Therapy Trial for Canavan Disease

Myrtelle Inc. announced encouraging interim results from its Phase 1/2 clinical trial of rAAV-Olig001-ASPA (MYR-101), an investigational gene therapy ...

Candel Therapeutics to Present CAN-2409 Data at SITC 2025

Candel Therapeutics will present data from its enLIGHTEN™ Discovery Platform and CAN-2409 program in non-small cell lung cancer (NSCLC) at the SITC 20...

Invivyd Announces U.S. IND Clearance and FDA Alignment for VYD2311 COVID Prevention Program

Invivyd announced that the FDA has cleared its IND application and provided feedback to advance its REVOLUTION clinical program for VYD2311, a vaccine...

DATROWAY® Improves Overall and Progression-Free Survival in Metastatic Triple-Negative Breast Cancer

AstraZeneca and Daiichi Sankyo's DATROWAY® (datopotamab deruxtecan-dlnk) demonstrated statistically significant and clinically meaningful improvements...

Marker Therapeutics Announces First Patient Treated in Off-the-Shelf Program for AML and MDS

Marker Therapeutics announced the treatment of the first patient in their Phase 1 RAPID study, evaluating MT-401, an off-the-shelf (OTS) multi-antigen...

Abivax Announces Positive Results from ABTECT Induction Trials of Obefazimod in Ulcerative Colitis Patients

Abivax announced late-breaking presentation of 8-Week ABTECT Induction Trial results in participants with and without prior inadequate response to adv...

Bolt Biotherapeutics Updates on BDC-4182 and Extends Cash Runway into 2027

Bolt Biotherapeutics announced an update on its Phase 1 dose escalation study of BDC-4182, an ISAC targeting claudin 18.2 for cancer treatment. Initia...

EG 427 Announces Positive Topline Results for EG110A in Neurogenic Bladder Patients

EG 427 announced positive initial results from a clinical trial of EG110A, a novel DNA therapy for neurogenic bladder. The study showed an over 88% re...

Lisata Therapeutics Announces Positive Preliminary Results from CENDIFOX Trial in Pancreatic Cancer

Lisata Therapeutics announced encouraging preliminary data from the Phase 1/2a CENDIFOX trial evaluating certepetide in combination with FOLFIRINOX fo...

CeleCor Therapeutics Announces Positive Phase 3 Results for Disaggpro in Heart Attack Patients

CeleCor Therapeutics announced positive topline results from its Phase 3 CeleBrate trial evaluating Disaggpro™ (zalunfiban) for heart attack treatment...

OS Therapies to Present at BioFuture 2025 and Discuss OST-HER2 Clinical Development

OS Therapies Inc. announces its participation in the BioFuture 2025 conference, where it will present an overview of the company with a focus on the c...

ONWARD Medical Announces Positive Results for ARC-IM Therapy in Blood Pressure Regulation after Spinal Cord Injury

ONWARD Medical N.V. announced concurrent publications in Nature and Nature Medicine highlighting the benefits of its investigational ARC-IM System for...

Skyhawk Therapeutics Announces Positive Interim Phase 1 Results for SKY-0515 in Huntington's Disease

Skyhawk Therapeutics announced positive interim results from its Phase 1 clinical trial of SKY-0515 for Huntington's disease (HD). The trial showed do...

Palvella Therapeutics Announces Publication on Rapamycin for Venous Malformations

Palvella Therapeutics announces a publication in Lymphatic Research and Biology highlighting advances in understanding venous malformation pathogenesi...

Protagonist Announces Clinical Data Presentations for Icotrokinra and PN-881 at EADV 2025 Congress

Protagonist Therapeutics announced new clinical data for Icotrokinra in plaque psoriasis from Phase 3 ICONIC-ADVANCE and ICONIC-LEAD studies, being pr...

Atossa Therapeutics Seeks Accelerated Approval for Low-Dose (Z)-Endoxifen for Breast Cancer Risk Reduction

Atossa Therapeutics has requested a Type C meeting with the FDA to discuss a regulatory strategy for accelerating the development of low-dose (Z)-endo...

CASI Pharmaceuticals Announces Dose Escalation in Phase 1 CID-103 ITP Study

CASI Pharmaceuticals announced that the Safety Monitoring Committee (SMC) recommended dose escalation of CID-103, an anti-CD38 monoclonal antibody, to...

Alto Neuroscience Validates EEG Biomarker for Schizophrenia, Strengthening Phase 2 Trial of ALTO-101

Alto Neuroscience announced positive results from an independent, prospective replication study validating theta-band inter-trial coherence (ITC) as a...

Positive Phase 1/2 Data for IDE397 and Trodelvy Combination in MTAP-Deletion Urothelial Cancer

IDEAYA Biosciences announced positive data from a Phase 1/2 trial evaluating the combination of IDE397, a MAT2A inhibitor, and Trodelvy (sacituzumab g...

Positive Phase 1 Data for IDE849 (SHR-4849) in Small Cell Lung Cancer

IDEAYA Biosciences and Hengrui Pharma presented positive Phase 1 data for IDE849 (SHR-4849), a DLL3-targeting TOP1 antibody-drug conjugate (ADC), at t...

Oncolytics Biotech Highlights Pelareorep Efficacy in Metastatic Colorectal Cancer

Oncolytics Biotech announced positive clinical and translational data for pelareorep in metastatic colorectal cancer (mCRC). In the REO 022 trial, pe...

RYBREVANT® and LAZCLUZE® Combination Prevents Acquired Resistance in First-Line EGFR-Mutated NSCLC

Johnson & Johnson announced new analyses from the Phase 3 MARIPOSA study demonstrating that the combination of RYBREVANT® (amivantamab-vmjw) and LAZCL...

iMDx Enrolls First Patient in GraftAssureDx Clinical Trial

Insight Molecular Diagnostics (iMDx) announced the enrollment of the first patient in its clinical trial for GraftAssureDx, a kitted organ transplant ...

Regeneron's Positive Phase 3 Allergy Trials Advance First-in-Class Antibody Blockers

Regeneron Pharmaceuticals announced positive results from two Phase 3 trials evaluating its first-in-class allergen-blocking antibodies for cat and bi...

Rapport Therapeutics to Announce Topline Phase 2a Results for RAP-219 in Focal Onset Seizures

Rapport Therapeutics will release topline data from its Phase 2a clinical trial of RAP-219 in patients with drug-resistant focal onset seizures on Sep...

SCYNEXIS Presents Data on Second-Generation Antifungal SCY-247 at TIMM-12

SCYNEXIS announced multiple presentations at the 12th Congress on Trends in Medical Mycology (TIMM-12) highlighting data on its second-generation anti...

Valitor Presents Preclinical Data on VLTR-559 for Wet AMD

Valitor announced positive preclinical data for VLTR-559, a long-acting anti-VEGF drug candidate for wet age-related macular degeneration (AMD). A no...

Gain Therapeutics Receives Approval for Phase 1b Dosing Extension of GT-02287 for Parkinson's Disease

Gain Therapeutics announced Australian approval to extend the Phase 1b clinical trial of its drug candidate, GT-02287, for Parkinson's disease by nine...

Clene Presents Preclinical Data Supporting CNM-Au8 for Parkinson's Disease

Clene Inc. announced positive preclinical data for CNM-Au8 in treating Parkinson's disease (PD). A novel neuronal model demonstrated CNM-Au8's abilit...

AL-S Pharma Announces Positive Phase 2 Results for AP-101 in ALS Treatment

AL-S Pharma reported positive topline results from its Phase 2 clinical trial of AP-101, a first-in-class monoclonal antibody targeting misfolded SOD1...

Sanofi's Amlitelimab Shows Promise in Phase 3 Atopic Dermatitis Study

Sanofi announced positive topline results from the COAST 1 Phase 3 clinical trial of amlitelimab, a monoclonal antibody targeting OX40L, in adults and...

Aspire Biopharma Announces Positive Final Results for Sublingual Aspirin in Treating Heart Attacks

Aspire Biopharma announced positive final results from a clinical trial evaluating its investigational sublingual aspirin product for suspected acute ...

Personalis' NeXT Personal MRD Test Shows Superiority in Neoadjuvant Lung Cancer Trial

Personalis, Inc. announced positive data from AstraZeneca's phase 3 NeoADAURA trial, demonstrating that their NeXT Personal molecular residual disease...

Zymeworks Discontinues ZW171 Clinical Development

Zymeworks Inc. announced the discontinuation of clinical development for ZW171, a T cell engager targeting gynecological, thoracic, and digestive syst...

Compass Pathways Announces Positive Phase 2 Results for COMP360 Psilocybin in PTSD

Compass Pathways announced the publication of positive Phase 2 results for their investigational drug COMP360, a synthetic psilocybin, in treating pos...

Ascletis to Present ASC30 Data at EASD Annual Meeting

Ascletis Pharma Inc. announced that it will present data from a 28-day multiple ascending dose study of its oral small molecule GLP-1 receptor (GLP-1R...

Merck's Enlicitide Decanoate Meets Primary Endpoints in Phase 3 Hypercholesterolemia Trial

Merck announced positive topline results from the Phase 3 CORALreef Lipids trial evaluating enlicitide decanoate, an investigational oral PCSK9 inhibi...

Olezarsen Shows Significant Reduction in Triglycerides and Acute Pancreatitis in sHTG Patients

Ionis Pharmaceuticals announced positive topline results from pivotal Phase 3 CORE and CORE2 studies of olezarsen in patients with severe hypertriglyc...

Baxdrostat Shows Significant Blood Pressure Reduction in Phase III Hypertension Trial

Positive results from the BaxHTN Phase III trial demonstrated that baxdrostat, a highly selective aldosterone synthase inhibitor, significantly reduce...

Alnylam to Advance Zilebesiran into Global Phase 3 Cardiovascular Outcomes Trial

Alnylam Pharmaceuticals announced plans to initiate a Phase 3 cardiovascular outcomes trial (CVOT) for zilebesiran, an investigational RNAi therapeuti...

Roche and Alnylam Advance Zilebesiran into Phase III Cardiovascular Outcomes Trial for Uncontrolled Hypertension

Roche and Alnylam announced the initiation of a Phase III cardiovascular outcomes trial (CVOT) for zilebesiran, an RNAi therapeutic, to reduce major a...

Merck's VERQUVO (vericiguat) Shows Mixed Results in Chronic Heart Failure Trials

Merck announced results from the Phase 3 VICTOR trial and a pooled analysis of VICTOR and VICTORIA trials evaluating VERQUVO (vericiguat) in chronic h...

Tourmaline Bio Presents Positive Phase 2 Data for Pacibekitug at ESC Congress 2025

Tourmaline Bio announced positive data from the ongoing Phase 2 TRANQUILITY trial of pacibekitug, a long-acting anti-IL-6 monoclonal antibody, present...

Telomir-1: A Novel Epigenetic Therapy Targeting DNA Methylation Pathways

Telomir Pharmaceuticals announced in vitro data supporting the potential of Telomir-1, a first-in-class epigenetic therapy, in treating cancer, aging,...

Zevra Therapeutics to Present Multiple Datasets on MIPLYFFA® (arimoclomol) at ICIEM

Zevra Therapeutics announced that four posters on MIPLYFFA® (arimoclomol), its approved therapy for Niemann-Pick disease type C (NPC), will be present...

Breath Diagnostics' OneBreath™ Technology Predicts Pneumonia Onset Through Exhaled Breath Analysis

Breath Diagnostics announced results from a peer-reviewed clinical study published in The Journal of Thoracic and Cardiovascular Surgery, demonstratin...

Airiver Medical Receives FDA IDE Approval for Pivotal Clinical Study of Airiver Pulmonary Drug Coated Balloon

Airiver Medical announced that it has received Investigational Device Exemption (IDE) approval from the FDA to conduct a pivotal clinical trial of its...

Actuate Therapeutics' Elraglusib Shows Promising Results in Metastatic Pancreatic Cancer

Actuate Therapeutics announced positive Phase 2 results for elraglusib (elra) in combination with gemcitabine and nab-paclitaxel (GnP) for metastatic ...

Coya Therapeutics Announces FDA Acceptance of IND for COYA 302 to Treat ALS

Coya Therapeutics announced that the FDA has accepted its Investigational New Drug (IND) application for COYA 302, a biologic combination therapy for ...

Immuneering and Lilly Partner to Evaluate Atebimetinib and Olomorasib in Phase 2 NSCLC Trial

Immuneering Corporation announced a clinical supply agreement with Eli Lilly and Company for olomorasib, to be used in combination with Immuneering's ...

ITF Therapeutics Announces Positive Long-Term Givinostat Data for Duchenne Muscular Dystrophy

ITF Therapeutics announced the publication of positive long-term safety and efficacy data for givinostat in treating Duchenne muscular dystrophy (DMD)...

Napo Pharmaceuticals to Meet with FDA Regarding Crofelemer for Ultrarare Pediatric MVID

Napo Pharmaceuticals, a Jaguar Health family company, plans to meet with the FDA to discuss the clinical development program for crofelemer in treatin...

MIRA Pharmaceuticals Completes Phase 1 Trial of Oral Ketamir-2 for Neuropathic Pain

MIRA Pharmaceuticals announced the successful completion of the single ascending dose (SAD) portion of its Phase 1 clinical trial for oral Ketamir-2, ...

Rocket Pharmaceuticals' Phase 2 Danon Disease Trial Resumes After FDA Lifts Clinical Hold

Rocket Pharmaceuticals announced that the FDA has lifted the clinical hold on its pivotal Phase 2 trial of RP-A501 for Danon disease. The hold was lif...

IDEAYA Biosciences Announces Agenda for 10-Year Anniversary R&D Day

IDEAYA Biosciences, Inc. announced the agenda for its 10-year anniversary R&D Day on September 8, 2025. The event will feature presentations on the c...

ZyVersa Therapeutics Highlights Data on Kidney Lipotoxicity in Diabetic Kidney Disease

ZyVersa Therapeutics announced data highlighting the critical need for therapies targeting kidney lipotoxicity in diabetic kidney disease (DKD). The ...

Largest-Ever Sickle Cell Disease Study Completed, Paving Way for New Treatments

Fifty1 AI Labs, in collaboration with The Gates Foundation and leading universities, has completed the largest-ever study of Sickle Cell Disease (SCD)...

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