Latest News
Keratin 8-Containing Complexes Could Be Attractive Therapeutic Targets in AATD
Patients With AATD May Not Recognize Symptom Changes as Exacerbations
Cigarette Smoke Exposure Stimulates Proinflammatory Macrophages in AATD
Biomarkers of Liver Stiffness Investigated in Pediatric Cholestatic Liver Diseases
Biomarkers Can Differentiate Mitochondrial Hepatopathies From Other Liver Diseases
Pharmacokinetics and Biochemical Efficacies Comparable Between 3 AATD Therapies
Long-Term Treatment of AATD With Alpha-1 MP Appears Safe
Rare Disease Patients and Advocate to Headline NORD Conference in October
Researchers Identify Factors Associated With COPD in Patients With AATD
AAT Secretion Is Cell-Type Dependent, Possibly Impacting AATD Treatment
New Recommendations for Managing Alpha-1 Antitrypsin Deficiency
New Allele Frequency Data Associated With AATD and CF
Latest Clinical Trials
Insights
The Challenge and Importance of Detecting AATD in Patients With Asthma
It is challenging for physicians to diagnose AATD in a patient with an existing asthma diagnosis since so many symptoms overlap.
Understanding the Disease Burden of AATD
Patients with alpha-1 antitrypsin deficiency, along with their caregivers, struggle with a significant and multifaceted disease burden.
The Practicality of At-Home Therapy for Treating AATD
Self-infusion is an option for patients with alpha-1 antitrypsin deficiency, meaning that the bulk of treatment can now take place at home.
Patient Perspectives
Weighing the Pros and Cons of AATD Patients Using Inhalers
If you had an alpha-1 antitrypsin deficiency (AATD) patient in front of you, would you prescribe inhalers?
My Encounter With COVID-19 as an AATD Patient
I got COVID-19 last week, so as an alpha-1 antitrypsin deficiency (AATD) patient, the whole experience is still fresh in my mind.
Music That Helps Me Deal With My AATD
There are specific songs that help me feel empowered as a person with alpha-1 antitrypsin deficiency (AATD).
The Challenges of Building Relationships While Managing AATD
For someone with alpha-1 antitrypsin deficiency (AATD), managing relationships can feel like being at sea during a storm.
Alpha-1 Features
The Alliance for Regenerative Medicine Fights for Better Cell, Gene Therapies
The Alliance for Regenerative Medicine aims to help in the development of safe and effective regenerative therapies that can benefit rare disease patients.
‘Fundamental Flaws’ Plague Drug Development Process, Ultragenyx CEO Dr. Emil Kakkis Says
Ultragenyx CEO Dr. Emil D. Kakkis said federal regulators should focus more on biomarkers and less on clinical endpoints when it comes to drug approvals.
Aytu BioPharma’s Healight Device Could Help Treat Rare Pulmonary Diseases
Aytu BioPharma has developed an endotracheal respiratory catheter which its CEO says has “tremendous potential” in IPF, PAH, and other respiratory diseases.
Inhaled Therapy Could Transform AATD Treatment Landscape
Israeli biopharma giant Kamada is banking that its inhaled AAT therapy will “revolutionize” the treatment of alpha-1 antitrypsin deficiency (AATD).
Rare Care Podcast
Israeli biopharma giant Kamada is banking that its inhaled AAT therapy will “revolutionize” the treatment of alpha-1 antitrypsin deficiency (AATD).
Mark Brantly, MD, scientific director of the Alpha-1 Foundation, is the vice-chair of research in the Department of Medicine at the University of Florida in Gainesville, and has spent most of his career researching a cure for alpha-1 antitrypsin deficiency (AATD).
Alpha-1 Meeting Coverage
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