Maria Pilar A. A.
- Country
- Spain
- Department
- Rheumatology Department
Research Overview
- Phase 3 Randomized, Double-Blind, Placebo-Controlled Study of ALXN1850 in Adolescents and Adults with Hypophosphatasia Naïve to Asfotase Alfa Treatment
- Phase 1/2 Multicenter, Open-label Study of Subcutaneous KK8123 in Adult Patients with X-linked Hypophosphatemia: Safety, Tolerability, and Pharmacokinetics Evaluation
Data & Insights
Has run more than one trial at 3 of 27 partner sites.
Specializations
Metabolic Bone Disorders
This investigator specializes in rare metabolic bone conditions, with particular focus on X-linked hypophosphatemia and hypophosphatasia. Their research encompasses innovative therapeutic approaches for these challenging disorders.
- X-linked Hypophosphatemia Treatment Development
- Novel Therapeutic Solutions for Hypophosphatasia
- Rare Metabolic Bone Disease Management
The investigator's work centers on evaluating new treatment options for patients with inherited metabolic bone disorders.
Clinical Development
Expertise in conducting first-in-human studies and phase 3 clinical trials, particularly focusing on novel therapeutic agents for rare genetic conditions affecting bone metabolism.
- Safety and Tolerability Assessment
- Pharmacokinetic Profiling
- Functional Outcome Evaluation
Their research encompasses both early-phase human studies and advanced clinical trials for therapeutic validation.
Therapeutic Innovation
The investigator evaluates novel therapeutic compounds including KK8123 and ALXN1850, focusing on their potential to address unmet medical needs in rare bone disorders.
- Treatment-Naive Patient Studies
- Subcutaneous Administration Methods
- Comparative Treatment Analysis
Their work involves assessing innovative therapeutic approaches for patients who have not received previous treatments.
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