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RAPID trial extension study finds augmentation therapy slows disease progression, supports early intervention

RAPID trial extension study finds augmentation therapy slows disease progression, supports early intervention
Friday, December 2, 2016
The medical journal The Lancet Respiratory Medicine on Thursday, Dec. 1, published findings of the RAPID Open Label Extension study of augmentation therapy for patients with emphysema and Alpha-1 Antitrypsin Deficiency. N. Gerard McElvaney, MD, lead author The original RAPID trial, sponsored by CSL Behring, randomly assigned 180 Alphas with emphysema to receive either...

House overwhelmingly passes 21st Century Cures bill

Thursday, December 1, 2016
WASHINGTON — The House overwhelmingly passed a far-reaching measure on Wednesday to increase funding for research into cancer and other diseases, address weaknesses in the nation’s mental health systems and help combat the prescription...

Arrowhead halts clinical trial of Alpha-1 liver drug candidate, plans to move ahead with subcutaneous AAT program

Wednesday, November 30, 2016
Arrowhead Pharmaceuticals has halted clinical trials of several potential drug therapies using its EX1 delivery system, including ARC-AAT, which had been showing good results in clinical trials to treat Alpha-1 liver disease, the company said in a...

Investigational drug showed deep reduction of alpha-1 protein in healthy volunteers, Arrowhead reports at Liver Meeting

Monday, November 14, 2016
Arrowhead Pharmaceuticals, Inc. delivered a poster presentation with Phase 1 clinical data and an oral presentation with preclinical data on ARC-AAT, its investigational medicine for the treatment of liver disease associated with Alpha-1...

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...

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...

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