Alpha 1 Antitrypsin Deficiency News and Research

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Exosome isolation using ultracentrifugation

In the outpatient clinic and lab, our research studies aim to understand biological functions of exosomes and membrane particles.

From Beckman Coulter Life Sciences - Centrifugation 9 Feb 2017

RNA silencing technique developed by spinoff from UM-Madison effective in battling hepatitis B

A method for "silencing" RNA that emerged from a University of Wisconsin-Madison spinoff company is in clinical trials in Europe, Asia and the United States against hepatitis B, an infection that can destroy the liver.

11 Aug 2016

New lung-targeted gene therapy shows promise in improving treatment for emphysema

Researchers have developed a new strategy using lung-targeted gene therapy that may lead to improved treatments for inherited diseases including emphysema.

6 Jul 2016

Innovative approach to treating AAT deficiency

Researchers have demonstrated the feasibility of delivering an RNA that encodes for the protein alpha-1-antitrypsin (AAT)--which is missing or nonfunctional in the genetic disorder AAT deficiency--into cells in the laboratory, enabling the cells to produce highly functional AAT.

28 Jul 2015

Therapy for genetically-caused emphysema slows progression of lung disease

A landmark clinical study in the Lancet provides convincing evidence that a frequently overlooked therapy for genetically-caused emphysema is effective and slows the progression of lung disease.

29 May 2015

Alnylam files clinical trial application for alpha-1 liver disease, presents data at DDW

Alnylam Pharmaceuticals, Inc. has announced that it has filed a Clinical Trial Application (CTA) with the U.K. Medicines and Healthcare products Regulatory Agency (MHRA) to initiate a Phase 1/2 clinical trial with ALN-AAT, a subcutaneously administered investigational RNAi therapeutic targeting alpha-1 antitrypsin (AAT) for the treatment of AAT deficiency-associated liver disease (alpha-1 liver disease).

19 May 2015

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