Clinicians Weigh in on When Augmentation Therapy Is Called for in AATD
Today, experts are increasingly recognizing the positive role that ordinary physicians can play in refining diagnostic and management guidelines.
AATD Insights
AATD Insights, CAD Insights, DMD Insights, Insights, LAL-D Insights, LCFAOD Insights, MG Insights, MTC Insights, SCD Insights
The Far-Reaching Impact of Innovative Clinical Trial Designs in Rare Disease
We examine the lasting impact of innovative clinical trial designs for rare diseases on other clinical experiments.
The Manifestations of AATD: Beyond the Pulmonary Pathology
We explore the various pulmonary and extrapulmonary manifestations of AATD, and the role of AAT augmentation therapy in both sets of symptoms.
Precision Medicine Becoming Increasingly Important in Treating AATD
We discuss the increasingly prevalent role of precision medicine in treating genetic diseases such as alpha-1 antitrypsin deficiency (AATD).
Examining the Pathological Effects of AATD on the Liver
We explore the evidence on the pathological effects of AATD on the human liver, particularly in individuals with the Pi*ZZ mutation.
Addressing Protein Misfolding in AATD Could Lead to Improved Therapies
Protein misfolding and its consequences are a key mechanism in the pathophysiology of alpha-1 antitrypsin deficiency.
Protease Inhibitors Could Aid in the Treatment of AATD
We examine how protease activity injures the lungs in patients with AATD, and whether protease inhibitors could be the therapeutic solution.
The Role of Next-Generation Sequencing in Diagnosing Infants With Cholestasis
We explore the role of next-generation sequencing in providing a genetic diagnosis for infants with a cholestatic disease, such as Alagille syndrome or AATD.
The Feasibility of Self-Administering AAT Therapy During the COVID-19 Pandemic
We explore how AAT therapy can be self-administered safely by AATD patients, which is vital when their ability to travel is limited.
Caring for Patients With AATD in the Context of COVID-19
We explore AATD care in the context of Covid-19, including a hypothesis that AAT therapy could ameliorate COVID-19 symptoms.
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