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FDA, Alpha-1 Foundation discuss placebos, trial of augmentation after lung transplants

FDA, Alpha-1 Foundation discuss placebos, trial of augmentation after lung transplants
Friday, September 30, 2016
Just over one year ago, members of the Alpha-1 community traveled from around the country to Silver Spring, Maryland for a meeting with the U.S. Food and Drug Administration (FDA).   Charlotte Mattison, right, gives emotional testimony about her condition at the FDA meeting with the Alpha-1 community in 2015. Over 200 Alphas, their caregivers and supporters filled the room at the...

PPTA statement says plasma protein products are safe, not affected by Zika virus

Saturday, September 24, 2016
The Plasma Protein Therapeutics Association (PPTA) has issued a statement saying that the safety of plasma protein therapies is not affected by the Zika virus (ZIKV). "According to recommendations from health authorities, measures such as...

Arrowhead begins Phase 2 trial of possible Alpha-1 liver treatment

Tuesday, September 6, 2016
California-based Arrowhead Pharmaceuticals Inc. announced Tuesday that it has begun a Phase 2 clinical study of ARC-AAT, the company's potential treatment for liver disease in people with Alpha-1 Antitrypsin Deficiency (AATD), using the...

Kamada Phase 2 trial shows promising results for inhaled augmentation

Wednesday, August 31, 2016
Kamada has released the results of a clinical trial showing that inhaled augmentation produces higher levels of alpha-1 protein in the airways than traditional intravenous augmentation. Mark Brantly, MD The company says the study’s...

ATS editorial warns against unregulated stem cell “treatments”

Monday, August 22, 2016
The Annals of the ATS, a publication of the American Thoracic Society, has published an editorial that warns against the use of unregulated, expensive stem cell remedies to treat severe respiratory diseases. The editorial, written by Laertis...

UMass gets 5-year, $11 million research grant for Alpha-1 gene therapy

Thursday, August 11, 2016
The National Heart, Lung, and Blood Institute (NHLBI) has awarded an $11 million, five-year grant to the University of Massachusetts Medical School to study new approaches to Alpha-1 gene therapy. Members of the UMass program project grant...

Alpha-1 Foundation leaders go to The White House to advocate better forms of detection and treatment for Alphas

Thursday, July 14, 2016
WASHINGTON – Alpha-1 Foundation representatives traveled to The White House this week to advocate better forms of detection and treatment for Alpha-1 Antitrypsin Deficiency (Alpha-1) under President Barack Obama’s Precision Medicine...

New targeted gene therapy could lead to improved treatments for Alphas

Monday, July 11, 2016
Researchers at Boston University are examining whether delivering gene therapy directly to the lungs of mice could improve treatment for Alphas. Andrew Wilson, MD Current therapies add normal alpha-1 antitrypsin (AAT) protein into the...

Researchers show off “gene editing” technique to Alpha-1 support group

Friday, June 3, 2016
Researchers are looking for a way to genetically “edit” out Alpha-1 in much the same way that a writer corrects misspelled words, or a film editor cuts old frames and splices in new ones. Members of the Alpha-1 Mainers Support Group...

Pope calls for "empathy" in treating people with rare diseases

Tuesday, May 3, 2016
Pope Francis told participants in a Vatican health summit on Friday that a renewed sense of empathy should fuel their work and research, particularly regarding treatment for people who have rare diseases. Pope Francis “It is fundamentally...

The Alpha-1 Project extends funding for NIH-NCATS fellowship for a third year

Wednesday, April 13, 2016
The Alpha-1 Project has announced that it will extend funding for the postdoctoral fellowship at the National Institutes of Health’s (NIH) National Center for Advancing Translational Sciences (NCATS) for a third year. The extension follows...

Alpha-1-To-One magazine Spring issue in your mail now

Wednesday, March 30, 2016
The Spring 2016 issue of Alpha-1-To-One magazine has been mailed to well over 20,000 people with free subscriptions, and will soon be available online. This is the cover story, "In search of new therapies for Alpha-1,"  part of a...

Kamada applies to market inhaled Alpha-1 therapy in Europe

Tuesday, March 29, 2016
Kamada has submitted a Marketing Authorization Application with the European Medicines Agency (EMA), seeking permission to market its inhaled therapy for Alpha-1 Antitrypsin Deficiency in Europe. The EMA, the European equivalent of the U.S. Food...

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