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Clinical trial for liver disease treatment will follow orphan drug designation

Clinical trial for liver disease treatment will follow orphan drug designation
Wednesday, February 21, 2018
The US Food and Drug Administration (FDA) granted orphan drug designation to Arrowhead Pharmaceuticals’ ARO-AAT, their second-generation investigational medicine for the treatment of a rare genetic liver disease associated with Alpha-1 Antitrypsin Deficiency (Alpha-1). ARO-AAT is a subcutaneous injection of an RNA interference (RNAi) therapeutic candidate for patients with liver...
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Alpha-1 researchers use CRISPR technology to create the first mouse model of genetic emphysema

Friday, February 16, 2018
Humans have only one gene that codes for the Alpha-1 protein, but mice have up to six, which made it impossible for decades to create a mouse model of the disease. Now a group of scientists, headed by Dr. Christian Mueller, of the Horae Gene...
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Lancaster woman celebrates anniversary of double lung transplant

Wednesday, February 14, 2018
As the anniversary of her double lung transplant passed, Crystal Chilcote spent the day with her daughter getting lunch and shopping. While these may seem like normal, everyday tasks for the average person, simply walking to her mailbox was not an...
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FDA approves test for the genetic diagnosis of Alpha-1

Tuesday, February 13, 2018
Grifols, Barcelona, has received approval from the US Food and Drug Administration (FDA) for a new genetic test to detect Alpha-1 Antitrypsin Deficiency (Alpha-1). The authorization marks the first time FDA has approved a biological molecular test...
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Alpha and leader Richard (Dick) Bueker passes away

Tuesday, February 13, 2018
It is with a heavy heart that the Alpha-1 Foundation announces the passing of Richard (Dick) Armin Bueker, beloved member of the Alpha-1 Community for many years, in the morning of Sunday, February 11, 2018, while sleeping. He was 78. Dick was...
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New study offers insights on genetic indicators of COPD risk

Friday, February 9, 2018
Researchers have discovered that genetic variations in the anatomy of the lungs could serve as indicators to help identify people who have low, but stable, lung function early in life, and those who are particularly at risk for chronic obstructive...
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A life-changing e-mail leads to a personal health breakthrough

Monday, February 5, 2018
Staying up later than usual, high school math teacher Heather Toepfer decided to turn on news, without knowing that a single evening newscast in September 2016 would ignite a series of events and medical insights that she now believes have saved...
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Rare Disease Day keeps on promoting research

Friday, February 2, 2018
Rare Disease Day takes place on the last day of February each year. This year it will be on Wednesday, February 28. The main objective of the day is to raise awareness amongst the general population and decision-makers about rare diseases and...
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First John W. Walsh Translational Research Award granted

Wednesday, January 31, 2018
MIAMI/BOSTON, January 31, 2018 – The Alpha-1 Foundation celebrates the first granting of the John W. Walsh Translational Research Award in Alpha-1 Antitrypsin Deficiency, an honorable award named after John W. Walsh, co-founder of the...
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Alpha-1 Foundation welcomes a new COO

Monday, January 29, 2018
The Alpha-1 Foundation would like to announce the appointment of Mr. Eddie Gloria as the new Chief Operating Officer (COO). Mr. Gloria will be replacing our former COO Marcia Ritchie, who retired last December. We want to take this...
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The effects of air pollution on human health

Thursday, January 25, 2018
The immediate effects of air pollution are hard to ignore. Watery eyes, coughing and difficulty breathing are acute and common reactions. An estimated 92% of the world’s population live in areas with dangerous levels of air pollution and,...
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Alpha-1 gene therapy trial doses first patient, recruitment continues

Thursday, January 11, 2018
Adverum Biotechnologies, a clinical-stage gene therapy company targeting unmet medical needs in several diseases, is recruiting patients for a clinical trial for Alpha-1 Antitrypsin (A1AT) Deficiency, designed to evaluate the safety and protein...
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Spark Therapeutics explores payment options after revealing price tag for first gene therapy in the US

Thursday, January 4, 2018
Philadelphia based pharmaceutical company, Spark Therapeutics, confirmed conversations with the U.S. Centers of Medicare and Medicaid Services on a proposal that would allow for multi-year payment plans for its innovative treatment, Luxturna, the...
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