Drug Database
PT

PT-007

✓ Approved

Pediatrix Therapeutics · Unknown · Unknown

What is PT-007?

PT-007 is a unknown developed by Pediatrix Therapeutics. It is approved for therapeutic indications via unknown.

Drug Profile

CompanyPediatrix Therapeutics
Drug ClassUnknown
RouteUnknown
StatusApproved
Sources: ClinicalTrials.gov, Pediatrix Therapeutics

Therapeutic Indications

PT-007 is developed for 1 unique indication across 1 therapeutic area.

Therapeutic AreaConditionPhase
Congenital, familial and genetic disordersHereditary haemochromatosis✓ Approved

Related Research Articles

PubMedDrug safety2026-09-20

A Text-Mining Algorithm for Identifying Drug Pairs Potentially Involved in Prescribing Cascades: Development and Internal Validation Within the Prescribing Inappropriateness Assessment (PINA) Digital Tool.

Carollo Massimo M, Forti Anna A, Crisafulli Salvatore S, L'Abbate Luca L et al.

Prescribing cascades (PCs) occur when an adverse drug reaction (ADR) is intentionally or unintentionally treated with an additional medication, potentially perpetuating inappropriate polypharmacy. Digital tools that systematically support the identification of potential PCs during medication review are lacking. Here, we describe the development of an algorithm designed to identify drug pairs potentially compatible with PCs and evaluate its classification performance. The algorithm was developed as a component of the Prescribing INappropriateness Assessment (PINA) digital tool, a software platform designed to support clinicians in conducting medication reviews. The algorithm logic identifies potential PCs by detecting intersections between ADRs and therapeutic indications extracted from Italian Summaries of Product Characteristics (SmPCs) and encoded as Medical Dictionary for Regulatory Activities (MedDRA) terms using a natural language processing pipeline. Four algorithm configurations were tested, differing in the MedDRA semantic levels used for matching: (1) Preferred Terms (PTs) only; (2) PTs with semantic extension to High Level Terms (HLTs); (3) PTs with extension to Standardised MedDRA Queries (SMQs); and (4) PTs with combined HLT/SMQ extension. Performance was assessed against a reference standard comprising 69 literature-supported positive drug pairs and 138 randomly generated negative drug pairs in a 1:2 ratio. Sensitivity, specificity, positive predictive value (PPV), negative predictive value (NPV), accuracy, F1 score, and Youden's index were calculated. All models demonstrated excellent specificity (≥ 99%). Sensitivity increased with semantic expansion: 50.7% for PT-only matching, 73.9% for PT+HLT, 68.1% for PT+SMQ, and 85.5% for PT+HLT/SMQ. The combined HLT/SMQ extension model achieved the highest overall performance, with an accuracy of 94.7%, a PPV of 98.3%, an NPV of 93.2%, an F1 score of 91.4, and a Youden's index of 84.8. False-positive rates remained minimal across all models. The newly developed algorithm demonstrated high classification performance, particularly when PT-level matching was extended using both HLTs and SMQs. This MedDRA-driven approach based on regulatory data may represent a scalable and reproducible strategy for supporting structured medication review and facilitating the recognition and clinical evaluation of potential PCs.

PMID 42763348
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PubMedJournal of pediatric endocrinology & metabolism : JPEM2026-09-20

Anti-Müllerian hormone levels in girls with precocious puberty.

Hepokur Merve Nur MN, Çamaş Aşan Önder AÖ, Taniş Gözde G, Çiçek Burçin B et al.

We aimed to evaluate the relationship between anti-Müllerian hormone (AMH) levels in cases of central precocious puberty (CPP), premature adrenarche (PA), and isolated premature thelarche (IPT) and those of healthy controls (HC). A total of 153 girls (40 PA, 39 CPP, 39 IPT, and 35 HC) were included in the study. AMH levels were assessed using the ELISA method. The mean age of all participants was 7.19 ± 0.63 (5.0-8.0) years. Height SDS was higher in the puberty and puberty variant groups compared with the healthy group. This difference was found to be statistically significant between the CPP and HC groups. Obesity was present in 20 of all participants (13 %). AMH levels were 0.125 ± 0.154, 0.086 ± 0.072, 0.084 ± 0.074, and 0.069 ± ;0.060 ng/mL in PA, PT, CPP, and HC groups, respectively. Although AMH levels were higher in the PA group, there was no statistically significant difference among the four groups. A weak negative correlation was found between AMH levels and BMI SDS (body mass index standard deviation score). AMH levels do not differ between prepubertal girls and those diagnosed with central precocious puberty or a variant of puberty of the same age.

PMID 42763793
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PubMedJournal of shoulder and elbow arthroplasty2026-09-20

Lower Geriatric Nutritional Risk Index is associated with increased risk of acromial stress fracture following reverse total shoulder arthroplasty.

Stokes Leonard L, Polam Vidhur V, Sauvanell Yousy Martinez YM, Hoffman Tammy T et al.

Acromial and scapular spine stress fractures are serious complications following reverse total shoulder arthroplasty (rTSA), and patient-level risk factors may contribute to their development. The Geriatric Nutritional Risk Index (GNRI) is a validated marker of physiologic reserve. The purpose of this study was to evaluate the association between GNRI and post-operative stress fracture after rTSA. We hypothesized that lower GNRI would be associated with increased fracture risk. We performed a retrospective 1:2 age- and sex-matched case-control study of patients undergoing primary rTSA. Forty-five patients who developed post-operative acromial or scapular spine stress fracture were matched to 90 controls without fracture. Demographic variables collected included age, sex, body mass index, diagnosis, and comorbidities. GNRI was calculated pre-operatively and post-operatively and analyzed as a continuous variable and using predefined thresholds (<98, <103, <109). Conditional logistic regression adjusted for diagnosis and comorbidities. Receiver-operating characteristic analysis assessed discriminatory ability. A total of 135 patients were included (mean age, 72.8 years; 53% female). Patients with stress fracture had lower pre-operative GNRI (107.4 ± 12.4 vs. 114.0 ± 13.9; P = .007) and post-operative GNRI (105.5 ± 14.9 vs. 114.8 ± 14.5; P = .001). Post-operative GNRI <109 was more common among fracture patients (60% vs. 30%; P = .001). In matched multivariable analysis, each 1-point increase in post-operative GNRI was associated with a 5% reduction in fracture odds (odds ratioOR, 0.95; 95% confidence interval [CI], 0.92-0.99; P = .015). Post-operative GNRI <109 was independently associated with fracture (odds ratio, 3.64; 95% CI, 1.02-13.02; P = .047). Receiver-operating characteristic analysis demonstrated moderate discrimination (area under the curve, 0.70; 95% CI, 0.60-0.79). In this matched case-control analysis, lower post-operative GNRI was independently associated with increased risk of acromial and scapular spine stress fracture following rTSA. GNRI may serve as a practical perioperative risk stratification tool in patients undergoing reverse shoulder arthroplasty.

PMID 42763527
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PubMedRSC chemical biology2026-09-19

Pt(iv) scaffold effects on nucleolar stress and DNA damage response pathways.

Griffin Christopher R CR, DeRose Victoria J VJ

The platinum compounds cisplatin and oxaliplatin are widely used anticancer agents. Although platinum compounds have long been thought to induce cell death through the DNA damage response (DDR), subsequent work has shown that oxaliplatin instead engages a nucleolar stress pathway characterized by disruptions to ribosome biogenesis. Importantly, tuning platinum compounds' non-labile ligand identity, particularly incorporation of the diaminocyclohexane (DACH) ligand, influences nucleolar stress induction. While effective, Pt(ii) drugs are limited by severe side effects arising, in part, from multiple targets as well as a lack of selectivity between malignant and non-malignant cells. The axial ligands of Pt(iv) compounds afford tunable properties that may improve selectivity. Here, we investigate how platinum compound ligand identity and oxidation state together dictate engagement of either the DDR or nucleolar stress pathway. Using a series of dicarboxylate Pt(iv) complexes derived from cisplatin and DACH-Pt scaffolds, we find that Pt(iv) compounds bearing aliphatic dicarboxylate axial ligands preserve the pathway specificity encoded by their Pt(ii) cores: cisplatin analogs primarily activate the DDR, whereas DACH-Pt analogs preferentially induce nucleolar stress. Increasing compound lipophilicity enhances antiproliferative activity and cellular accumulation, and accelerates pathway activation. However, analysis of uptake, response onset, and IC50 values reveals that lipophilicity alone does not fully account for biological activity. Notably, we identify the most potent nucleolar stress inducer reported, a Pt(iv) compound requiring ∼1000-fold lower concentrations to trigger nucleolar stress than the DACH-Pt(ii) analogue. Together, these findings establish Pt(iv) ligand design as a strategy to optimize antiproliferative activity while selectively intensifying DDR or nucleolar stress.

PMID 42761964
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PubMedJournal of ISAKOS : joint disorders & orthopaedic sports medicine2026-09-19

Quality-of-life outcomes and osteoarthritis development at 10-years after anterior cruciate ligament reconstruction: A large randomized clinical trial comparing patellar tendon, hamstring tendon and double-bundle grafts.

Mohtadi Nicholas N, Chan Denise D, Hunter Dana D, Bertiche Pablo P et al.

This large randomized clinical trial compared disease-specific quality-of-life, clinical and radiographic outcomes 10-years following Anterior Cruciate Ligament (ACL) reconstruction with patellar tendon (PT), single-bundle (HT; quadruple-stranded semitendinosis-gracilis/tendons), or double-bundle (DB; 2-stranded semitendinosis and 2-stranded gracilis tendon) hamstring tendon constructs. Three-hundred-and-thirty ACL deficient patients (183 males; 14-50 years) were randomized to PT (n=110, mean 28.7 years), single-bundle HT (n=110, mean 28.5 years), or double-bundle DB, (n=110, mean 28.3 years). An independent assessor and patients were blinded to treatment allocation up to 2-years. Outcomes were assessed at Baseline, 2-, 5- and 10-years post-operatively. ACL Quality-of-Life (ACL-QOL) score. pivot shift, kneeling pain, International Knee Documentation Committee (IKDC) scores, Tegner Activity Level, traumatic re-injuries and contralateral ACL tears. A blinded assessor graded each knee compartment using the International Knee Documentation Committee (IKDC) radiographic scale. Two-hundred-and-eighty-seven patients (87%) completed 10-year follow-up (mean 10.2 1.2 years). Mean ACL-QOL scores increased over time for all groups (p<0.001), with no statistically significant difference at 10-years (PT=76.4 21.0 (95%CI 72.1-80.7); HT=77.7 20.5 (95%CI 73.4-81.9); DB=77.3 21.5 (95%CI 73.0-81.7); p=0.912). Fewer traumatic re-injuries occurred with PT grafts (PT=8%; HT=22%; DB=21%, p=0.023). No statistically significant differences for contralateral ACL tears (PT=13%; HT=13%; DB=10%; p=0.801), kneeling pain (PT=6%; HT=4%; DB=7%; p=0.637), Normal/Nearly Normal IKDC Objective scores (PT=73%; HT=68%; DB=72%; p=0.724) or the remaining secondary outcomes. Pivot shift grade ≥2 was less likely with a PT reconstruction (PT=18%; HT=25%; DB=24%; p=0.584). Forty-five percent of patients had radiographic OA at 10-year follow-up (medial=17%; lateral=29%; patellofemoral=6%). ACL-QOL scores were statistically significantly lower for patients with medial (69.3 3.2) and lateral OA (72.8 2.4). The PT group had more lateral OA (PT=42%, HT=23%, DB=23%; p=0.010), but no statistically significant differences in medial (PT=14%, HT=12%, DB=24%, p=0.094) or patellofemoral OA (PT=5%, HT=7%, DB=8%, p=0.782). Contributors of medial OA included baseline age, medial meniscectomy, chondral damage and subsequent secondary surgery. Contributors of lateral OA included PT graft, valgus alignment and lateral meniscectomy. ConclusionsACL reconstructive surgery consistently improves quality-of-life, with most patients remaining highly active at 10 years. ACL-QOL scores were lower for patients with medial and lateral OA. More traumatic re-injuries occurred with HT and DB reconstructions. PT reconstructions were more likely to have lateral radiographic changes. All effect sizes demonstrating these associations were small. Level 1 CLINICAL TRIAL REGISTRATION: ClinicalTrials.gov # ID number.

PMID 42762953
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PubMedIndian pacing and electrophysiology journal2026-09-19

Early Intraoperative Risk Stratification of Persistent High Pacing Threshold After Atrial Septal Pacing With a Lumenless 3830 Lead: A Pilot Study.

Hayasaka Kazuto K, Shirai Yasuhiro Y, Sasaki Takeshi T, Shimosato Hikaru H et al.

Atrial septal pacing with the 3830 lead is increasingly used, but some patients show high pacing thresholds (PT) immediately after fixation, and elevated thresholds may persist. Practical criteria based on early intraoperative findings for identifying such cases have not been established. We retrospectively studied 53 consecutive patients who underwent cardiac implantable electronic device implantation with the 3830 lead positioned in the high right atrial septum. PT, sensing amplitude, impedance, and current of injury (COI) were measured immediately after fixation, at 5 and 10 min after fixation, post-procedure, at 1 week, and 3 months. The initial high-threshold group was defined as PT ≥ 1.5 V/0.4ms immediately after fixation. In this group, we evaluated a two-step strategy using PT and COI at 5 min. The primary outcome was PT > 1.0 V/0.4ms at 1 week. Of 53 patients, 26 were classified into the initial high-threshold group. In this group, PT improved from 2.000[1.750-2.438] V immediately after fixation to 0.750[0.500-0.875] V at 1 week (p<0.001). Three patients (11.5%) met the primary outcome. PT ≥ 2.5 V/0.4ms at 5 min yielded 100.0% sensitivity, 95.7% specificity, 75.0% positive predictive value, and 100.0% negative predictive value. In this cohort, adding a 5-min COI cutoff ≤ 2.1 mV to 5-min PT yielded 100.0% for all diagnostic metrics in this cohort. In this pilot study, most cases with high PT immediately after fixation improved within 1 week. A two-step assessment using PT and COI at 5 min after fixation may provide an exploratory framework for intraoperatively stratifying cases at risk of persistent high threshold at 1 week.

PMID 42762893
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