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

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. [More]
New RNA aptamer can prevent pathogenic protein misfolding

New RNA aptamer can prevent pathogenic protein misfolding

Several diseases occur when mutations cause misfolding of proteins. These include "serpinopathies" which is a group of rare heritable diseases. They are caused by mutations of so-called "serpin" inhibitors of proteolytic enzymes involved in blood coagulation, tissue remodeling, and other important physiological functions. [More]
Innovative approach to treating AAT deficiency

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. [More]
Therapy for genetically-caused emphysema slows progression of lung disease

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

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). [More]
New Breathe NIOV System uses Bayer’s injection-molded Makrolon polycarbonate

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

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. [More]
Johns Hopkins scientists use genome editing technology to alter human stem cells

Johns Hopkins scientists use genome editing technology to alter human stem cells

A powerful “genome editing” technology known as CRISPR has been used by researchers since 2012 to trim, disrupt, replace or add to sequences of an organism’s DNA. [More]
TSRI study examines body’s own response against chronic protein misfolding

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. [More]
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 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]
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