Medical Tidbits, Vertex Receives Australian Approval for ORKAMBI(R) (lumacaftor/ivacaftor), the First Medicine to Treat the Underlying Cause of Cystic Fibrosis in People Ages 12 and Older with Two Copies of the F508del Mutation

breaking medical news, research for cancer, cliinical research, FDA approvals

LONDON–(BUSINESS WIRE)–Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced that the Therapeutic Goods Administration (TGA) of Australia has approved ORKAMBI® 200/125 (lumacaftor 200mg and ivacaftor 125mg), the first medicine to treat the underlying cause of cystic fibrosis (CF) in people ages 12 and older who have two copies of the F508del mutation. In Australia, there are approximately 1,000 people with CF ages 12 and older who have two copies of this mutation. The Australian reimbursement process for ORKAMBI is already underway as part of the parallel regulatory and reimbursement processes between the TGA and the Pharmaceutical Benefits Advisory Committee (PBAC).  read more

New discoveries on the connection between nicotine and type 2 diabetes

Researchers at Lund University in Sweden have made two new discoveries with regard to the beta cells’ ability to release insulin. The findings can also provide a possible explanation as to why smokers have an increased risk of type 2 diabetes.

The study was conducted on mice and donated beta cells from humans, and is now published in the scientific journal Cell Report s . read more

A new paradigm for end of life care

A recent report from seven charities estimated 48,000 people who died last year experienced poor care when dying. In her new book, The Bell Lap, author Muriel Murch lays the ground for caregivers to reach beyond the confines of the settings of illness, whether home, hospital, or long-term care and become active and involved participants in the world of the patient. read more

Sarepta Therapeutics Announces Long Term Plan to Consolidate Facilities within Massachusetts

Sarepta Therapeutics, Inc. (NASDAQ:SRPT), a developer of innovative RNA-targeted therapeutics, today announced a long term plan to move all of the Company’s operations to Massachusetts, as part of a strategic plan to increase operational efficiency. read more

SciFluor Life Sciences Awarded Second U.S. Patent for Integrin αvβ3 (SF0166) Inhibitors Designed for the Topical Treatment of Retinal Disease

CAMBRIDGE, Mass.–(BUSINESS WIRE)–SciFluor Life Sciences, Inc., an emerging clinical stage biopharmaceutical company that strives to create innovative best-in-class therapeutics for patients in multiple disease categories including ophthalmologic and neurologic disease, announced today that the U.S. Patent and Trademark Office (USPTO) has issued U.S. Patent No. 9,266,884 with claims covering the use of the novel compound SF0166. In December 2014, the U.S. Patent and Trademark Office (USPTO) issued U.S. Patent No. 8,901,144 with claims covering the composition of matter of SciFluor’s SF0166, a small molecule integrin antagonist. read more

Pfizer Commences $5 Billion Accelerated Share Repurchase

NEW YORK–(BUSINESS WIRE)–Pfizer Inc. (NYSE:PFE) today announced that it has entered into an accelerated share repurchase agreement with Goldman, Sachs & Co. (GS&Co.) to repurchase $5 billion of Pfizer’s common stock. This agreement is part of Pfizer’s existing share repurchase authorization. read more

Epizyme Unveils Corporate Strategy to Guide Efforts over Next Five Years

CAMBRIDGE, Mass.–(BUSINESS WIRE)–Epizyme, Inc., (NASDAQ:EPZM) a clinical stage biopharmaceutical company creating novel epigenetic therapies for patients with cancer, today announced its corporate vision and strategy through 2020. During the next five years, Epizyme will focus on four transformative activities: read more

Early HPV vaccination provides best protection

The HPV vaccine is most effective against high-grade cervical lesions if given before the age of 17, according to a new register-based study from Karolinska Institutet and the Public Health Agency of Sweden. The results, which are published in the  International Journal of Cancer , show that the vaccine is effective at preventing high-grade lesions. read more

Thorn Medical PLC Granted Approval for Human Stem Cell Research and Treatment by Bahamas Ministry of Health

Thorn Medical PLC (“Thorn” or the “Company”), the international healthcare group, today announces that it has received full approval from the Bahamas Ministry of Health to carry out stem cell research and treatment on humans.The approval allows Thorn to use human stem cells to cure or treat the full range of conditions and illnesses that humans are prone to. For further information, please visit Thorn Medical’s website

Results of Open-Label Trial for CRESEMBA® (isavuconazonium sulfate) Treatment of Invasive Mucormycosis Published in The Lancet Infectious Diseases  

PRNewswire/ — Astellas today announced that results from the Phase 3 VITAL trial evaluating CRESEMBA® (isavuconazonium sulfate) in adult patients with invasive mucormycosis were published in The Lancet Infectious Diseases. The article, titled “Isavuconazole treatment for mucormycosis: a single-arm open-label trial and case-control analysis,” appears in the March 8, 2016 online issue and will appear in a future print issue of The Lancet Infectious Diseases.  read more



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