Alpha 1 Antitrypsin Deficiency News and Research RSS Feed - Alpha 1 Antitrypsin Deficiency News and Research

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

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). [More]
Kamada to present Phase 2/3 clinical trial results of AAT to treat alpha-1 antitrypsin deficiency

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. [More]
FDA grants clearance for Breathe Technologies’ Non-Invasive Open Ventilation System

FDA grants clearance for Breathe Technologies’ Non-Invasive Open Ventilation System

Breathe Technologies, Inc. announced today that the U.S. Food and Drug Administration has granted the fifth 510(k) clearance for its Non-Invasive Open Ventilation System, allowing its use with compressed air supply for non-oxygen dependent patients. [More]
Baxter plans to create two independent global healthcare companies

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

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. [More]
Drug used to treat schizophrenia, dementia has potential for treating liver disease

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. [More]
UMMS researchers commissioned by Alpha-1 Project to develop PiZ antibody

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). [More]
IQWiG publishes new clinical practice guidelines for treating people with COPD

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. [More]
Polyphor, Roche agree to develop and commercialize antibiotic for bacterial infections

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. [More]
New smartphone app for patients with hemophilia and related bleeding disorders

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. [More]
A1-PI treatment slows progression of emphysema in patients with AATD

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. [More]
Scientists identify new mutation in a gene that causes Alpha-1 Antitrypsin Deficiency

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. [More]
ALN RNAi therapeutic program represents novel approach for treatment of AAT deficiency

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. [More]
Oxidative stress occurs in genetic models of alpha-1-antitrypsin deficiency

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. [More]
SLU professor to investigate natural history and progression of Alpha-1 Antitrypsin Deficiency

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. [More]
GSK, University of Cambridge aim to discover and develop new medicines for liver disease

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. [More]

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. [More]
RCSI study: Alpha-1 antitrypsin deficiency more prevalent in Ireland

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. [More]
Nature publishes Sangamo's gene correction strategy for A1AT deficiency

Nature publishes Sangamo's gene correction strategy for A1AT deficiency

Sangamo BioSciences, Inc. (Nasdaq: SGMO) announced the publication of a preclinical study demonstrating highly specific, functional correction of the alpha 1-antitrypsin (A1AT) gene defect in patient-derived induced pluripotent stem cells (iPSCs) using zinc finger nucleases. [More]
Isis designates ISIS-AATRx candidate in collaboration with GSK

Isis designates ISIS-AATRx candidate in collaboration with GSK

Isis Pharmaceuticals, Inc. announced today that it has designated the second development candidate, ISIS-AATRx, in its collaboration with GlaxoSmithKline [More]