Alpha 1 Antitrypsin Deficiency News and Research

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New Breathe NIOV System uses Bayer’s injection-molded Makrolon polycarbonate

Imagine not being able to climb the stairs without stopping to take a break, or getting winded trying to stand and do the dishes. Unfortunately these are common realities for individuals suffering from respiratory conditions. Ventilators can help reduce the work of breathing by unloading the ancillary respiratory muscles, but they are often bulky and heavy, creating additional limitations for users. The Breathe Non-Invasive Open Ventilation (NIOV) System technology is a better solution.

13 Feb 2015

Dr. Hans Clevers receives ISSCR's McEwen Award for Innovation

The International Society for Stem Cell Research has awarded Dr. Hans Clevers, senior author on two important papers published recently in the scientific journal Cell, the society's McEwen Award for Innovation.

23 Jan 2015

TSRI study examines body’s own response against chronic protein misfolding

“Protein misfolding” diseases such as cystic fibrosis and Alzheimer’s may be seriously exacerbated by the body’s own response against that misfolding, according to a new study led by scientists at The Scripps Research Institute.

20 Nov 2014

Kamada announces top-line results from Phase II/III trial for treatment of inherited emphysema

Kamada Ltd., a plasma-derived protein therapeutics company focused on orphan indications, announces preliminary top-line results from the Phase II/III pivotal clinical trial in Europe and Canada of the Company's proprietary inhaled Alpha-1 Antitrypsin (AAT) therapy for the treatment of Alpha-1 Antitrypsin Deficiency (AATD or inherited emphysema).

16 May 2014

Kamada to present Phase 2/3 clinical trial results of AAT to treat alpha-1 antitrypsin deficiency

Kamada Ltd., a plasma-derived protein therapeutics company focused on orphan indications, will announce results from its Phase 2/3 clinical trial of its proprietary inhaled alpha-1 antitrypsin (AAT) to treat alpha-1 antitrypsin deficiency (AATD) in Europe and Canada on Friday, May 16, 2014, at approximately 7:30 a.m. Eastern time.

15 May 2014

Baxter plans to create two independent global healthcare companies

Baxter International Inc. today announced plans to create two separate, independent global healthcare companies -- one focused on developing and marketing innovative biopharmaceuticals and the other on life-saving medical products. Both will be global leaders in their respective markets.

From Baxter International Inc. 27 Mar 2014

SLU researchers receive $1.4M grant to study alpha-1 antitrypsin deficiency in adults

Researchers at Saint Louis University will study alpha-1 antitrypsin deficiency in adults, an inherited disease that can cause liver damage, to answer various questions that could lead to effective methods of treatment.

27 Mar 2014

Drug used to treat schizophrenia, dementia has potential for treating liver disease

Opening up a can of worms is a good way to start hunting for new drugs, recommend researchers from Children's Hospital of Pittsburgh of UPMC and the University of Pittsburgh School of Medicine.

4 Feb 2014

UMMS researchers commissioned by Alpha-1 Project to develop PiZ antibody

Researchers at the University of Massachusetts Medical School (UMMS) have been commissioned by the Alpha-1 Project (TAP) to develop a PiZ antibody. The antibody will be used to track the presence of mutant alpha-1 PiZ protein in human blood serum, an essential tool in testing potential therapies for Alpha-1 Antitrypsin Deficiency (Alpha-1).

13 Jan 2014

IQWiG publishes new clinical practice guidelines for treating people with COPD

On 3 January 2014 the German Institute for Quality and Efficiency in Health Care published the results of a literature search for evidence-based clinical practice guidelines on the treatment of people with chronic obstructive pulmonary disease.

9 Jan 2014

Polyphor, Roche agree to develop and commercialize antibiotic for bacterial infections

Polyphor Ltd, a privately held pharmaceutical company, and Roche announced today that they have entered into an exclusive worldwide license agreement to develop and commercialize Polyphor's investigational macrocycle antibiotic, POL7080, for patients suffering from bacterial infections caused by Pseudomonas aeruginosa.

4 Nov 2013

New smartphone app for patients with hemophilia and related bleeding disorders

BioRx, a national specialty pharmacy and infusion services provider, announced the launch of its industry's first smartphone app designed for patients with hemophilia and related bleeding disorders.

23 Oct 2013

A1-PI treatment slows progression of emphysema in patients with AATD

Treatment with an Alpha-1 proteinase inhibitor (A1-PI), a naturally occurring protein that protects lung tissue from breakdown and protects the lung's elasticity, is effective in slowing the progression of emphysema in patients with Alpha-1 antitrypsin deficiency (AATD), a life-threatening genetic disorder, according to a new study presented at the 2013 American Thoracic Society International Conference.

22 May 2013

Scientists identify new mutation in a gene that causes Alpha-1 Antitrypsin Deficiency

Scientists have identified a new mutation in the gene that causes the inherited disease known as Alpha-1 Antitrypsin Deficiency (Alpha-1), which affects roughly one in 2,500 people of European descent.

14 Dec 2012

ALN RNAi therapeutic program represents novel approach for treatment of AAT deficiency

Alnylam Pharmaceuticals, Inc., a leading RNAi therapeutics company, announced today that it has presented new pre-clinical data with an RNAi therapeutic targeting alpha-1 antitrypsin (AAT) for the treatment of liver disease associated with AAT deficiency. These data were presented at the 63rd Annual Meeting of the American Association for the Study of Liver Diseases (AASLD, "The Liver Meeting") held November 9-13, 2012 in Boston.

15 Nov 2012

Oxidative stress occurs in genetic models of alpha-1-antitrypsin deficiency

A team of researchers under the direction of Dr. Jeffrey Teckman in the Department of Pediatrics at St. Louis University, have demonstrated that oxidative stress occurs in a genetic model of alpha-1-antitrypsin deficiency.

30 Oct 2012

SLU professor to investigate natural history and progression of Alpha-1 Antitrypsin Deficiency

Jeffrey Teckman, M.D., professor of pediatrics and biochemistry and molecular biology at Saint Louis University, has received a $1.4 million grant from the Alpha-1 Foundation to investigate the natural history and progression of Alpha-1 Antitrypsin Deficiency, an inherited disease that can cause liver damage in children as well as adults.

4 Oct 2012

GSK, University of Cambridge aim to discover and develop new medicines for liver disease

A new collaboration based at the University of Cambridge will aim to discover and develop new medicines to treat liver disease.

10 Sep 2012

COPDF announces expansion of Bronchiectasis Research Registry

The COPD Foundation (COPDF) today announced expansion of its Bronchiectasis Research Registry to include patients with Nontuberculous Mycobacteria (NTM), an often misdiagnosed orphan disease that can cause severe lung infections that mimic tuberculosis when certain individuals are exposed to soil and water.

12 Jan 2012

RCSI study: Alpha-1 antitrypsin deficiency more prevalent in Ireland

Researchers from the Royal College of Surgeons in Ireland (RCSI), Beaumont Hospital, and Trinity College Dublin have conducted a study which has found that Ireland has one of the highest incidences in the world of a genetic condition that causes severe hereditary emphysema.

20 Oct 2011