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Study: Music therapy improves COPD symptoms

Friday, January 15, 2016
A new study has found that patients with chronic obstructive pulmonary disease (COPD) and other chronic respiratory conditions showed signs of improvement in symptoms, psychological well-being, and quality of life when exposed to music therapy in...

Foundation congratulates Califf for Senate Committee's unanimous approval of his nomination as FDA commissioner

Tuesday, January 12, 2016
The Alpha-1 Foundation on Tuesday congratulated Robert Califf, MD, on the Senate Health, Education, Labor, and Pensions (HELP) Committee’s unanimous approval of his nomination to be the U.S. Food and Drug Administration (FDA)...

Alpha-1 Foundation scientific, clinical directors edit book on Alpha-1 for clinicians and researchers

Tuesday, January 12, 2016
A new book edited by two prominent Alpha-1 experts explores the causes and symptoms of the condition, and highlights the role of healthcare organizations in providing treatment. Geared toward pulmonary clinicians and scientists, “Alpha-1...

Arrowhead’s ARC-AAT granted EMA orphan drug designation

Monday, January 11, 2016
PASADENA, Calif. — Arrowhead Research Corporation, a biopharmaceutical company that is developing targeted gene therapies, today announced that the European Medicines Agency (EMA) has granted Orphan Drug Designation to ARC-AAT, a treatment...

Skyrocketing drug prices leave cures out of reach for some patients

Thursday, January 7, 2016
Sophisticated drugs are opening the door, scientists say, to an era of "precision medicine." They're also ushering in an age of astronomical prices. New cancer drugs are routinely priced at more than $100,000 a year — nearly twice the...

FDA approved 21 new drugs to treat rare "orphan" diseases in 2015

Tuesday, January 5, 2016
The U.S. Food and Drug Administration (FDA) announced this week that it approved 21 new drugs to treat rare or “orphan” diseases in 2015, nearly half (47 percent) of all novel new drugs that were approved for the year. This...

Why do some Alphas develop liver disease?

Tuesday, December 15, 2015
In Alphas with the ZZ genotype, most of the alpha-1 protein made in the liver misfolds and gets trapped in the liver cells instead of moving normally into the bloodstream. Because of the ZZ gene mutation, the misfolded proteins link to one...

Webinar will answer questions about natural history studies (like Alpha-1 Adult Liver Study)

Wednesday, December 9, 2015
Alphas and others with rare diseases are invited to join a free webinar on natural history studies for rare diseases at 11 a.m. EST on Thursday, Dec. 10. "If there is one area in which the rare disease patient can make a contribution to rare...

Alpha-1-To-One magazine Winter issue in your mail now

Thursday, December 3, 2015
The Winter 2015 issue of Alpha-1-To-One magazine is in the mail to some 27,000 people with free subscriptions and is now available online. Read the cover story, "A New Beginning: Frank Willersinn gets second chance at life and makes the most...

November is Alpha-1 Awareness Month

Monday, November 2, 2015
MIAMI, Nov. 2, 2015 – The Alpha-1 Foundation is marking Alpha-1 Awareness Month, designed to increase knowledge about Alpha-1 Antitrypsin Deficiency (Alpha-1). Throughout the month of November, people with Alpha-1 will hold educational...

Buyer beware: Alphas urged to avoid companies advertising unproven stem cell "treatments" that can cost you a fortune

Wednesday, October 28, 2015
This is a note from the Alpha-1 Foundation explaining why Alphas should be wary of claims advertised on the Internet by companies claiming benefits from unproven stem cell and other cell-based "therapies." What are stem cells? Stem cells are the...

FDA launches learning tool on how to bring new drugs to market

Thursday, October 22, 2015
The U.S. Food and Drug Administration (FDA) has launched a new learning tool designed to help patients, patient advocacy groups, health professionals, small businesses, pharmaceutical and clinical innovators learn more about the drug regulatory...

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