Hypophosphatasia Understanding the Disease and Improving Diagnosis

Slides:



Advertisements
Similar presentations
Phase Difference = Phase Difference = 0.05.
Advertisements

Messmer, E M The Pathophysiology, Diagnosis, and Treatment of Dry Eye Disease Dtsch Arztebl Int 2015; 112(5): 71-82; DOI: /arztebl
Strensiq ® - Asfotase alfa Manufacturer: Alexion FDA Approval Date: October 23, 2015.
US Burden of Disease and Injury Study, 1996
Asfotase Alfa Drugbank ID : DB09105.
Radiographs of the right femur in a patient with juvenile Paget disease before (left) and after (right) 10 months of treatment with calcitonin. Note the.
The Nurse View: Management of Pancreatic Cancer
Perspectives on Managing Polycythemia Vera for the Nurse Practitioner and Physician Assistant.
Understanding Treatment Goals for Patients With Onychomycosis
Treatment List: Case # { } {List treatment and tooth numbers}
Harrison CN et al. Proc ASH 2015;Abstract 59.
New Horizons for SMA.
Overview The Burden of COPD Main Challenges in the Diagnosis of COPD.
Endocrine Combinations in HR-Positive MBC: The Future Is Now
IBS-D -- From Diagnosis to Management
An Interprofessional Approach to Optimizing Patient Care in Allergy
Cholesterol, CV Events, and PCSK9 Inhibitors Highlights From the AHA
Program Goals. Out of the Periphery and Into Comprehensive Care: Managing Diabetic Peripheral Neuropathy.
Advancing Management of Anemia With the Latest Data From ERA-EDTA 2018
Diabetes and Dyslipidemia
Introduction. Looking Beyond HIV Infection: CCR5 Blockade as an Immunomodulatory Concept 
Patient Presentation. Patient Presentation PRIMO Study Incidence of Muscle Adverse Effects.
This talk will follow the topical structure suggested by the ABP:
Renal and Extrarenal Complications of ADPKD: Facilitating Better Patient Care.
Seasonal Allergic Rhinitis
Hunter Syndrome: Why We Need to Diagnose and Treat Early
Can Primary Care Physicians Take the Lead in Combating Obesity?
Statin Selection in Special Patient Populations: A Case Challenge
New Treatments for CTEPH
The Parkinson's Disease Psychosis Journal Club
A Comprehensive Strategy Toward the Patient With Rhinorrhea
Updates in Preventing Skeletal-Related Events in Multiple Myeloma
The future of urate-lowering strategies for gout
Multidisciplinary Perspectives on Interstitial Lung Diseases
Opportunities and Challenges in the Management of Advanced STS
Hyperhidrosis Is Burdensome!
Atopic Dermatitis. Introduction to a Clinical Course On New Developments In Atopic Dermatitis.
Program Goals. Program Goals Introduction Causes of Hypoparathyroidism.
Immunotherapy for cSCC
Program Goals. Program Goals Introduction Causes of Hypoparathyroidism.
ADVANCED PARKINSON'S DISEASE:
Pseudobulbar Affect or Psychiatric Condition?
Improving Outcomes in Psoriatic Arthritis
CMV in the HSCT Recipient
Treatment-Resistant Schizophrenia
Battling the Flu: Latest Advances in the Influenza Treatment Arsenal
Case 1 Presentation Case 1 (cont) Assessment and Recommendations.
Implications of Emerging Treatments for Beta-Thalassemia
Hepatitis C: After the Diagnosis
Challenges in Severe Asthma
Diabetes Pandemic. The Economics of Managing Patients With Diabetes and Coronary Artery Disease: A Payer Perspective.
Managing Age-Related Clinical Issues in Hemophilia
EGPA.
Program Goals Disclaimers Defining Refractory Seizures.
Are We Making Progress in the Management of Huntington Disease?
Update on the Management of Atopic Dermatitis
Introduction. Emerging Treatments for Thrombocytopenia in Adults With CLD Requiring Elective Invasive Procedures.
Elevated Admission Plasma Glucose Following ACS
A Multidisciplinary Perspective on the Management of Hypoparathyroidism.
An Unmet Need.
Disease Burden of VTE Phases of VTE Treatment.
Optimizing Outcomes for Patients With Systemic Lupus Erythematosus: Key Considerations.
At the Crossroads of Coagulation
Name that bone....
Evaluating Success of Current Treatments for HCM
NAFLD and NASH in Europe and Canada
The Real Burden of Rosacea
CKD Is a Global Burden With Major Implications
Management of NTM Lung Disease
Diagnosing HFpEF: What Every Cardiologist Needs to Know
Presentation transcript:

Hypophosphatasia Understanding the Disease and Improving Diagnosis

Program Goals

Patient Case Referral for Premature Tooth Loss

Suspicions of HPP

Skeletal Survey

Radiographs

What is HPP?

Mechanism of HPP

Pathophysiology in Bone

Why is Vitamin B6 Elevated? HPP Pathophysiology in the CNS

HPP Common Presentation

Perinatal HPP

Infantile HPP

Childhood HPP

Adult HPP

Odontohypophosphatasia

HPP Disease Burden

Diagnosis of HPP

Diagnosis ALP Levels

Diagnosis Vitamin B6/PLP/PPi/PEA Levels

HPP Treatment

HPP Treatment Challenges

HPP Current Treatment: Asfotase Alfa

Asfotase Alfa Infantile Phase 2 Trial

Infantile Phase 2 Trial Results

Infantile Phase 2 Trial Safety

Patient Case Follow-up

Mortality Rates For Perinatal and Juvenile HPP

Asfotase Alfa Perinatal or Juvenile HPP

Asfotase Alfa Juvenile Trial

Juvenile Trial Results

Juvenile Trial Safety

Closing Comments

Abbreviations