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157 Diseases | | 20 Products |
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112 Trials |  |
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- || Retrospective data, Review, Journal, BRCA Biomarker, PARP Biomarker, Metastases: Poly (ADP-ribose) Polymerase Inhibitors Have Comparable Efficacy with Platinum Chemotherapy in Patients with BRCA-positive Metastatic Castration-resistant Prostate Cancer. A Systematic Review and Meta-analysis. (Pubmed Central) - May 17, 2024
Moreover, we found that PARPi and platinum therapy were comparable in terms of PSA50 response rate and OS, highlighting that platinum is a valid treatment option for BRCA-positive mCRPC patients. However, prospective interventional studies comparing these agents are essential to provide a higher level of evidence.
- | CB-103 / Cellestia Biotech, Naglazyme (galsulfase) / AnGes MG, BioMarin
Journal: Unveiling the role of novel carbohydrate-binding modules in laminarin interaction of multimodular proteins from marine Bacteroidota during phytoplankton blooms. (Pubmed Central) - May 17, 2024
Exploration of bacterial metagenome-assembled genomes (MAGs) from phytoplankton blooms in the North Sea showed that both laminarin-binding CBM families are widespread among marine Bacteroidota. The high protein abundance of CBM102- and CBM103-containing proteins during phytoplankton blooms further emphasizes their significance in marine laminarin utilization.
- || Xtandi (enzalutamide capsule) / Pfizer, Astellas, Talzenna (talazoparib) / Pfizer
Clinical, FDA event, Journal, Combination therapy, BRCA Biomarker, PARP Biomarker, Metastases: US Food and Drug Administration Approval Summary: Talazoparib in Combination With Enzalutamide for Treatment of Patients With Homologous Recombination Repair Gene-Mutated Metastatic Castration-Resistant Prostate Cancer. (Pubmed Central) - May 15, 2024
Approval was therefore limited to patients with HRRm mCRPC, for whom there was a statistically significant and clinically meaningful improvement in rPFS and favorable OS results. This represents the first approval for the first-line treatment of patients with HRRm mCRPC.
- | RGFP966 / BioMarin
Journal, Epigenetic controller: Histone deacetylase-3 regulates the expression of the amyloid precursor protein and its inhibition promotes neuroregenerative pathways in Alzheimer's disease models. (Pubmed Central) - May 11, 2024
For this purpose, N2a mouse neuroblastoma cells as well as organotypic brain cultures (OBCSs) of 5XFAD and wild-type mice were incubated with various concentrations of the HDAC3 selective inhibitor RGFP966 (0.1-10??M) for 24?h...mRNA sequencing analysis revealed that HDAC3 inhibition modulated neuronal regenerative pathways related to neurogenesis, differentiation, axonogenesis, and dendritic spine density in OBCSs. Our findings highlight the complexity and diversity of the effects of HDAC3 inhibition on AD models and suggest that HDAC3 may have multiple roles in the regulation of APP expression and processing, as well as in the modulation of neuroinflammatory and neuroprotective genes.
- || Review, Journal, Tumor mutational burden, PARP Biomarker, PD(L)-1 Biomarker, IO biomarker: A rare case of TFEB/6p21/VEGFA-amplified renal cell carcinoma diagnosed by whole-exome sequencing: clinicopathological and genetic feature report and literature review. (Pubmed Central) - May 11, 2024
Our findings highlight the complexity and diversity of the effects of HDAC3 inhibition on AD models and suggest that HDAC3 may have multiple roles in the regulation of APP expression and processing, as well as in the modulation of neuroinflammatory and neuroprotective genes. We clarified the patient's challenging diagnosis and discussed the clinicopathology, immunophenotype, differential diagnosis, and molecular genetic information regarding TFEB/6p21/VEGFA-amplified RCC via exome analysis and a literature review.
- |||| Aldurazyme (laronidase) / BioMarin, Sanofi
New P3 trial: Efficacy and Safety of P046 (Laronidase-CinnaGen) Compared to Aldurazyme (clinicaltrials.gov) - May 9, 2024
P3, N=12, Completed, Sponsor: Cinnagen
- MODULE 4: New Considerations with the Use of PARP Inhibitors for Metastatic Castration-Resistant Prostate Cancer (mCRPC) (Hemisfair Ballroom 2) - May 8, 2024 - Abstract #AUA2024AUA_4316;
We clarified the patient's challenging diagnosis and discussed the clinicopathology, immunophenotype, differential diagnosis, and molecular genetic information regarding TFEB/6p21/VEGFA-amplified RCC via exome analysis and a literature review. This activity is CME-accredited by Research To Practice and supported by educational grants from Astellas and Pfizer Inc, AstraZeneca Pharmaceuticals LP, Bayer HealthCare Pharmaceuticals, Janssen Biotech Inc, administered by Janssen Scientific Affairs LLC, and Merck.
- Combination strategies to overcome radioresistance in metastatic Castration-Resistant Prostate Cancer () - May 8, 2024 - Abstract #SNMMI2024SNMMI_1582;
This activity is CME-accredited by Research To Practice and supported by educational grants from Astellas and Pfizer Inc, AstraZeneca Pharmaceuticals LP, Bayer HealthCare Pharmaceuticals, Janssen Biotech Inc, administered by Janssen Scientific Affairs LLC, and Merck. Purpose/Background: Radiopharmaceutical therapy (RPT) with [177Lu]Lu-PSMA-617 (Pluvicto
- Talzenna (talazoparib) / Pfizer
StarPEG nanocarriers: A versatile nanoplatform for tumor imaging and therapy () - May 8, 2024 - Abstract #SNMMI2024SNMMI_1498;
The 177Lu-starPEG-DOTA1-ACUPA3 demonstrated PSMA-targeted uptake and internalization in PSMA+ cells and xenografts. Thus, 89Zr-surrogates may be employed as agents to predict drug delivery and correlate with therapeutic outcomes.
- | Palynziq (pegvaliase-pqpz) / BioMarin
Journal, Real-world evidence, Real-world: Pegvaliase therapy for phenylketonuria: Real-world case series and clinical insights. (Pubmed Central) - May 6, 2024
Despite potential challenges, pegvaliase therapy represents a substantial promise in managing PKU in Italy. Patient education, personalized treatment approaches, and careful monitoring are important to ensure optimal patient outcomes.
- | Trodelvy (sacituzumab govitecan-hziy) / Gilead
Journal: Antibody Drug Conjugate Sacituzumab Govitecan Enables A Sequential TOP1/PARP Inhibitor Cancer Therapy Strategy in Breast Cancer Patients. (Pubmed Central) - May 6, 2024
P1/2
While SG dosed concurrently with talazoparib is not tolerated clinically due to an insufficient therapeutic window, sequential dosing of SG then talazoparib proved a viable strategy. These findings support further clinical development of the combination and suggest that ADC-based therapy may facilitate novel, mechanism-based dosing strategies.
- | RGFP966 / BioMarin
Journal, IO biomarker, Epigenetic controller: Histone deacetylase 3-specific inhibitor RGFP966 attenuates oxidative stress and inflammation after traumatic brain injury by activating the Nrf2 pathway. (Pubmed Central) - May 6, 2024
Overall, our findings demonstrate that RGFP966 can mitigate the first brain damage and neurological impairments in TBI. The underlying mechanism involves triggering the Nrf2-mediated AO system and negatively regulating the HMGB1/TLR4-mediated NLRP3 inflammasome pathway.
- | Talzenna (talazoparib) / Pfizer, Vonjo (pacritinib) / SOBI
Enrollment open, Combination therapy: Pacritinib w/ Talazoparib in Pts w/ Myeloproliferative Neoplasms Unresponsive to JAK2 Inhibition (clinicaltrials.gov) - May 3, 2024
P1, N=24, Recruiting, Sponsor: Fox Chase Cancer Center
The underlying mechanism involves triggering the Nrf2-mediated AO system and negatively regulating the HMGB1/TLR4-mediated NLRP3 inflammasome pathway. Not yet recruiting --> Recruiting
- ||| Palynziq (pegvaliase-pqpz) / BioMarin
P3 data, Journal: Pegvaliase for the treatment of phenylketonuria: Final results of a long-term phase 3 clinical trial program. (Pubmed Central) - May 2, 2024
P3
Not yet recruiting --> Recruiting In conclusion, using data from three key pegvaliase clinical trials, participants treated with pegvaliase were able to reach clinically significant blood Phe reductions to clinical thresholds of ?600, ?360, or
- | Talzenna (talazoparib) / Pfizer, quisinostat (JNJ 26481585) / ChemRar, Viriom
Journal, Combination therapy: New synergistic combination therapy approaches with HDAC inhibitor quisinostat, cisplatin or PARP inhibitor talazoparib for urothelial carcinoma. (Pubmed Central) - May 2, 2024
These combinations appeared to be well tolerated in normal cells. In conclusion, our results suggest new promising combination regimes for treatment of UC, also in the cisplatin-resistant setting.
- | Journal, Metastases: What is the future of immune checkpoints inhibitors for metastatic triple negative breast cancers? (Pubmed Central) - Apr 30, 2024
In conclusion, our results suggest new promising combination regimes for treatment of UC, also in the cisplatin-resistant setting. No abstract available
- || Review, Journal: First-line combination treatment with PARP and androgen receptor-signaling inhibitors in HRR-deficient mCRPC: Applying clinical study findings to clinical practice in the United States. (Pubmed Central) - Apr 30, 2024
No abstract available Here, we review the newly approved PARPi plus ARSI treatments within the context of the mCRPC treatment landscape, provide an overview of practical considerations for the combinations in clinical practice, highlight the importance of HRR testing, and discuss the benefits of treatment intensification for patients with mCRPC.
- | Preclinical, Journal, PARP Biomarker: Curcumin Alone and Combined With PI3K Inhibitors Elicits Positive Effects on Oropharyngeal Cancer Cell Lines Regardless of HPV Status. (Pubmed Central) - Apr 28, 2024
Here, we review the newly approved PARPi plus ARSI treatments within the context of the mCRPC treatment landscape, provide an overview of practical considerations for the combinations in clinical practice, highlight the importance of HRR testing, and discuss the benefits of treatment intensification for patients with mCRPC. Curcumin alone led to dose-dependent responses and when combined with BYL719, positive effects were revealed, as they were when it was combined with MK-1775 or PD-0332991, suggesting a potential use of some of these combinations for HPV+ OPSCC.
- | Kuvan (sapropterin) / BioMarin, Daiichi Sankyo
Journal: Children and Adolescents with Early Treated Phenylketonuria: Cognitive Development and Fluctuations of Blood Phenylalanine Levels. (Pubmed Central) - Apr 27, 2024
Sapropterin dihydrochloride enhances Phe tolerance in patients with PKU. Increasing blood Phe fluctuations following enhanced Phe tolerance may indicate that the treatment not only allows patients to relax their Phe-restricted diet but also may support cognitive development in patients.
- | Talzenna (talazoparib) / Pfizer
Journal: Poly(ADP-Ribose) Polymerases-Inhibitor Talazoparib Inhibits Muscle Atrophy and Fatty Infiltration in a Tendon Release Infraspinatus Sheep Model: A Pilot Study. (Pubmed Central) - Apr 26, 2024
Finally, a significantly (p < 0.04) higher proportion and size of fast myosin heavy chain-II fiber type was observed in the treatment group. This study shows that PARP-inhibition with Talazoparib inhibits the progression of both muscle atrophy and fatty infiltration over 16 weeks in retracted sheep musculotendinous units.
- || Talzenna (talazoparib) / Pfizer, Tazverik (tazemetostat) / Eisai, Ipsen
Enrollment closed, Phase classification, Combination therapy, Metastases: Phase Ia/Ib Talazoparib + Tazemetostat for mCRPC (clinicaltrials.gov) - Apr 26, 2024
P1, N=35, Active, not recruiting, Sponsor: Dana-Farber Cancer Institute
This study shows that PARP-inhibition with Talazoparib inhibits the progression of both muscle atrophy and fatty infiltration over 16 weeks in retracted sheep musculotendinous units. Recruiting --> Active, not recruiting | Phase classification: P1a/1b --> P1
- | Bavencio (avelumab) / EMD Serono, Talzenna (talazoparib) / Pfizer
Trial completion date, Trial primary completion date, Metastases: TALAVE: Induction Talazoparib Followed by Combination of Talazoparib and Avelumab in Advanced Breast Cancer (clinicaltrials.gov) - Apr 24, 2024
P1/2, N=24, Active, not recruiting, Sponsor: Georgetown University
Recruiting --> Active, not recruiting | Phase classification: P1a/1b --> P1 Trial completion date: Mar 2024 --> Dec 2024 | Trial primary completion date: Mar 2024 --> Dec 2024
- || Voxzogo (vosoritide) / BioMarin
PK/PD data, Journal: Development of a Weight-Band Dosing Approach for Vosoritide in Children with Achondroplasia Using a Population Pharmacokinetic Model. (Pubmed Central) - Apr 23, 2024
P2, P3
Trial completion date: Mar 2024 --> Dec 2024 | Trial primary completion date: Mar 2024 --> Dec 2024 The weight-band dosing regimen reduced the number of recommended dose levels by body weight and is expected to simplify dosing for children with achondroplasia and their caregivers.
- | Lynparza (olaparib) / Merck (MSD), AstraZeneca
Journal, BRCA Biomarker, PARP Biomarker: Activated NAD+ biosynthesis pathway induces olaparib resistance in BRCA1 knockout pancreatic cancer cells. (Pubmed Central) - Apr 20, 2024
Upregulation of intracellular NAD+ levels by the addition of nicotinamide also induced resistance to olaparib and talazoparib in C1 cells. Taken together, our findings suggest that upregulation of intracellular NAD+ is one of the factors underlying the acquisition of PARP inhibitor resistance.
- Roctavian (valoctocogene roxaparvovec-rvox) / BioMarin
The Impact of a Cost Savings Cap on the Cost Effectiveness of Valoctocogene Roxaparvovec for the Treatment of Hemophilia A in the United States () - Apr 14, 2024 - Abstract #ISPOR2024ISPOR_2534;
This is the first model comparing the cost-effectiveness of ValRox versus emicizumab. The $150,000 per year cost cap was influential in projecting the cost-effectiveness of ValRox.
- | Roctavian (valoctocogene roxaparvovec-rvox) / BioMarin
Journal, Gene therapy: Three-year outcomes of valoctocogene roxaparvovec gene therapy for hemophilia A. (Pubmed Central) - Apr 13, 2024
The $150,000 per year cost cap was influential in projecting the cost-effectiveness of ValRox. Hemostatic efficacy was maintained, and safety remained unchanged from previous years.
- | PRO135 / BioMarin
Journal: Identification and characterization of the critical genes encoding Cd-induced enhancement of SOD isozymes activities in Zhe-Maidong (Ophiopogon japonicus). (Pubmed Central) - Apr 12, 2024
Hemostatic efficacy was maintained, and safety remained unchanged from previous years. The molecular docking experiments performed on the interaction between oxygen ion (O2
- || Keytruda (pembrolizumab) / Merck (MSD), Talzenna (talazoparib) / Pfizer, Tecentriq (atezolizumab) / Roche
Retrospective data, Review, Journal, BRCA Biomarker, PARP Biomarker, PD(L)-1 Biomarker, IO biomarker, Metastases: Efficacy and safety of first-line treatment for metastatic triple-negative breast cancer: A network meta-analysis. (Pubmed Central) - Apr 11, 2024
For PD-L1 and BRCA mutation-positive tumors, atezolizumab/pembrolizumab combined with nab-paclitaxel and talazoparib is an effective treatment option. Neutropenia, diarrhea, and fatigue are frequently occurring serious adverse events.
- Roctavian (valoctocogene roxaparvovec-rvox) / BioMarin
SAFETY AND EFFICACY OF VALOCTOCOGENE ROXAPARVOVEC GENE TRANSFER FOR SEVERE HEMOPHILIA A: AN UPDATE FROM 4 YEARS AFTER TREATMENT (Chicago Ballroom VII; Virtual) - Apr 9, 2024 - Abstract #THSNA2024THSNA_335;
P3
Neutropenia, diarrhea, and fatigue are frequently occurring serious adverse events. Return to prophylaxis was defined as
- Talzenna (talazoparib) / Pfizer
Talazoparib Plus Enzalutamide in Metastatic Castration-Resistant Prostate Cancer: Safety Analyses From the Randomized, Placebo-Controlled, Phase 3 TALAPRO-2 Study (Exhibit Hall) - Apr 7, 2024 - Abstract #HOPA2024HOPA_428;
To provide a detailed overview of the safety profile of talazoparib plus enzalutamide in the all-comers population of the TALAPRO-2 trial with the aim of understanding how adverse events were managed. Learning Objectives:
- Talzenna (talazoparib) / Pfizer
Exposure-Safety Analyses of Talazoparib in Combination With Enzalutamide in Patients with Metastatic Castration-Resistant Prostate Cancer (mCRPC) in TALAPRO-2 Trial (Exhibit Hall) - Apr 7, 2024 - Abstract #HOPA2024HOPA_398;
P3
Learning Objectives: To characterize the relationship between talazoparib exposure and selected safety endpoints including grade 3 and higher (Gr?3) anemia, neutropenia, and thrombocytopenia, and to identify potential prognostic factors for these safety endpoints in patients with mCRPC in the TALAPRO-2 study (NCT03395197) Learning Objectives:
- Evaluation of PARPi utilization for treatment of prostate cancer at St. Luke's Cancer Institute () - Apr 7, 2024 - Abstract #HOPA2024HOPA_244;
Olaparib has garnered an additional indication for homologous recombination repair (HRR) muted mCRPC in May 2023.1 Talazoparib in combination with enzalutamide was approved June 2023 for the treatment of HRR mutated mCRPC... Will be presented.
- | Talzenna (talazoparib) / Pfizer
Trial completion date, Trial primary completion date, Combination therapy: NAKIP-AML: Natural Killer Cell Immunotherapy in Combination With PARP-inhibition in Acute Myeloid Leukemia (clinicaltrials.gov) - Apr 4, 2024
P1/2, N=54, Not yet recruiting, Sponsor: German Cancer Research Center
Will be presented. Trial completion date: Jun 2027 --> Jun 2028 | Trial primary completion date: Jun 2027 --> Jun 2028
- Roctavian (valoctocogene roxaparvovec-rvox) / BioMarin
Phase classification: GENEr8-3: Study to Evaluate the Efficacy and Safety of Valoctocogene Roxaparvovec, With Prophylactic Steroids in Hemophilia A (clinicaltrials.gov) - Apr 4, 2024
P3, N=22, Active, not recruiting, Sponsor: BioMarin Pharmaceutical
Trial completion date: Jun 2027 --> Jun 2028 | Trial primary completion date: Jun 2027 --> Jun 2028 Phase classification: P3b --> P3
- Roctavian (valoctocogene roxaparvovec-rvox) / BioMarin
Health-related quality of life improvements following valoctocogene roxaparvovec gene therapy in people without bleeds or target joints at baseline: a post hoc analysis from GENER8-1 (Exhibit Hall (Hall 9); Virtual) - Apr 1, 2024 - Abstract #WFH2024WFH_970;
- || Trodelvy (sacituzumab govitecan-hziy) / Gilead, Talzenna (talazoparib) / Pfizer
Phase classification, Trial completion date, Trial primary completion date, Combination therapy, Metastases: Study to Evaluate Sacituzumab Govitecan in Combination With Talazoparib in Patients With Metastatic Breast Cancer. (clinicaltrials.gov) - Apr 1, 2024
P1/2, N=75, Recruiting, Sponsor: Massachusetts General Hospital
Phase classification: P3b --> P3 Phase classification: P1b/2 --> P1/2 | Trial completion date: Dec 2024 --> Dec 2026 | Trial primary completion date: Dec 2023 --> Dec 2025
- || Voxzogo (vosoritide) / BioMarin
Clinical data, Retrospective data, Review, Journal: Clinical outcomes and medical management of achondroplasia in Japanese children: A retrospective medical record review of clinical data. (Pubmed Central) - Mar 30, 2024
Before vosoritide approval in Japan, patients with ACH could start growth hormone (GH) treatment at age 3?years...The mean height at age 4?years with/without GH was 83.6/79.8?cm. These results improve our understanding of young patients with ACH in Japan and confirm that early diagnosis of ACH and monitoring of complications help facilitate appropriate interventions.
- | Talzenna (talazoparib) / Pfizer, Reblozyl (luspatercept-aamt) / BMS, Merck (MSD)
Japanese regulatory, Journal: PMDA regulatory update on approval of new drugs and revisions of precautions; approval of talazoparib tosilate for prostate and breast cancer, and luspatercept for myelodysplastic syndrome in Japan. (Pubmed Central) - Mar 29, 2024
These results improve our understanding of young patients with ACH in Japan and confirm that early diagnosis of ACH and monitoring of complications help facilitate appropriate interventions. No abstract available
- || Talzenna (talazoparib) / Pfizer, Mylotarg (gemtuzumab ozogamicin) / UCB, PDL, Pfizer
Enrollment open: Talazoparib and Gemtuzumab Ozogamicin for the Treatment of CD33 Positive Relapsed or Refractory Acute Myeloid Leukemia (clinicaltrials.gov) - Mar 27, 2024
P1, N=25, Recruiting, Sponsor: Roswell Park Cancer Institute
No abstract available Active, not recruiting --> Recruiting
- | Palynziq (pegvaliase-pqpz) / BioMarin
Journal: First successful outcomes of pegvaliase (PALYNZIQ) in children. (Pubmed Central) - Mar 26, 2024
This report addresses the potential broader applications of Pegvaliase in children, as well as its safety and tolerability in this age group. However, larger sample sizes and robust methodologies are required to validate such findings.
- | RGFP966 / BioMarin
Journal, Epigenetic controller: Critical Role of histone deacetylase 3 in the regulation of kidney inflammation and fibrosis. (Pubmed Central) - Mar 25, 2024
Thus, our study identified HDAC3 as a critical regulator of inflammation and fibrosis of the kidney through deacetylation of NF-?B unlocking its transcriptional activity. Hence, targeting HDAC3 could serve as a novel therapeutic strategy for CKD.
- | Brineura (cerliponase alfa) / BioMarin
Journal: Intravitreal enzyme replacement for inherited retinal diseases. (Pubmed Central) - Mar 25, 2024
The evidence suggests that IVT ERT with rhTPP1 may be a safe and effective treatment for CLN2 retinopathy. However, the optimal dosage and frequency to achieve the best possible outcomes requires further investigation as does patient selection.
- || Talzenna (talazoparib) / Pfizer, Inqovi (decitabine/cedazuridine) / Otsuka
Trial completion, Trial completion date, Trial primary completion date, Metastases: Study of ASTX727 Plus Talazoparib in Patients With Triple Negative or Hormone Resistant/HER2-negative Metastatic Breast Cancer (clinicaltrials.gov) - Mar 22, 2024
P1, N=34, Completed, Sponsor: Kathy Miller
However, the optimal dosage and frequency to achieve the best possible outcomes requires further investigation as does patient selection. Recruiting --> Completed | Trial completion date: Jul 2025 --> Mar 2024 | Trial primary completion date: Dec 2024 --> Jan 2024
- | Talzenna (talazoparib) / Pfizer
Trial completion date, Trial primary completion date, Metastases: Evaluation of Talazoparib, a PARP Inhibitor, in Patients With Somatic BRCA Mutant Metastatic Breast Cancer: Genotyping Based Clinical Trial (clinicaltrials.gov) - Mar 19, 2024
P2, N=30, Recruiting, Sponsor: Massachusetts General Hospital
Recruiting --> Completed | Trial completion date: Jul 2025 --> Mar 2024 | Trial primary completion date: Dec 2024 --> Jan 2024 Trial completion date: Jul 2024 --> Dec 2025 | Trial primary completion date: Dec 2023 --> Jul 2025
- | Kuvan (sapropterin) / BioMarin, Daiichi Sankyo
New P1 trial: Novel Approaches for Improving Vascular Function in Veterans With HFpEF (clinicaltrials.gov) - Mar 15, 2024
P1, N=90, Recruiting, Sponsor: D. Walter Wray
- | Talzenna (talazoparib) / Pfizer
Journal, PARP Biomarker, PD(L)-1 Biomarker, IO biomarker, Synthetic lethality: In Situ Formation of Fibronectin-Enriched Protein Corona on Epigenetic Nanocarrier for Enhanced Synthetic Lethal Therapy. (Pubmed Central) - Mar 14, 2024
To investigate the underlying mechanism, the tumor immune microenvironment and various gene expressions by RNAseq are explored. Moreover, the BMN/PAZA combination increased the immunogenicity and synergized with PD-1 antibody in improving the overall therapeutic effect in an orthotopic model of lung cancer (LLC).
- | Roctavian (valoctocogene roxaparvovec-rvox) / BioMarin
Clinical Trial,Phase II, Journal: Long-term safety and efficacy outcomes of valoctocogene roxaparvovec gene transfer up to 6 (Pubmed Central) - Mar 14, 2024
Moreover, the BMN/PAZA combination increased the immunogenicity and synergized with PD-1 antibody in improving the overall therapeutic effect in an orthotopic model of lung cancer (LLC). Valoctocogene roxaparvovec safety and efficacy profiles remain largely unchanged; genomic investigations showed no association with a parotid tumour.
- Therapeutic Resistance Models and Treatment Sequencing in Advanced Prostate Cancer (301B) - Mar 12, 2024 - Abstract #AUA2024AUA_3062;
e. , within NGATs or within taxanes, or within PARPi's).
- | Brineura (cerliponase alfa) / BioMarin
Journal: Classic and Atypical Late Infantile Neuronal Ceroid Lipofuscinosis in Latin America: Clinical and Genetic Aspects, and Treatment Outcome with Cerliponase Alfa. (Pubmed Central) - Mar 12, 2024
Follow-up after the initiation of cerliponase alfa showed slower progression or stabilization of the disease, associated with adequate clinical outcomes and stable functional scores. These improvements were consistent in both clinical phenotypes.
- | Kuvan (sapropterin) / BioMarin, Daiichi Sankyo
Journal: Maximal dietary responsiveness after tetrahydrobiopterin (BH4) in 19 phenylalanine hydroxylase deficiency patients: What super-responders can expect. (Pubmed Central) - Mar 12, 2024
This cohort demonstrates the maximally achievable dietary liberalization which some PKU patients may expect with BH4 therapy. Health benefits are considered to accrue in patients with increased intact protein.