Cystic Fibrosis News and Research

Latest Cystic Fibrosis News and Research

EMA's CHMP gives positive opinion for PTC Therapeutics' Translarna

PTC Therapeutics, Inc. today announced that following its request for re-examination, the Committee for Medicinal Products for Human Use of the European Medicines Agency has adopted a positive opinion regarding the company's application for a conditional marketing authorization of Translarna (ataluren) for the treatment of nonsense mutation Duchenne muscular dystrophy (nmDMD) in ambulatory patients aged five years and older.

23 May 2014

Aradigm receives QIDP designation for its inhaled antibiotic candidate, Pulmaquin

Aradigm Corporation (the "Company") today announced that the U.S. Food and Drug Administration (FDA) has designated the Company's lead inhaled antibiotic candidate, Pulmaquin®, as a Qualified Infectious Disease Product (QIDP).

20 May 2014

Study shows technology improves transition into adulthood for adolescents with chronic disease

Adolescents with chronic diseases (ACD), such as cystic fibrosis, gastrointestinal disorders (including Crohn's disease) and Type 1 diabetes, often find the transition of managing their health care needs into adulthood to be challenging.

19 May 2014

Aradigm reports total revenue of $6.6 million for Q1 2014

Aradigm Corporation today announced financial results for the first quarter and three months ended March 31, 2014.

14 May 2014

Trends, opportunities and threats in the life science industry: an interview with Bob Coughlin, CEO, MassBio

We were extremely excited to launch the Impact 2020 report at our annual meeting. Our five-year strategic plan is focused on value, early-stage financing and ensuring more patient-centered discussions.

5 May 2014

FDA approves Parion’s investigational new drug application for treatment of dry eye disease

Parion Sciences, a company dedicated to the development of novel treatments for ocular and pulmonary diseases, announced today that it has received acceptance from the U.S. Food and Drug Administration (FDA) of its Investigational New Drug (IND) application for P-321 Ophthalmic Solution for the treatment of dry eye disease.

2 May 2014

Vertex: Conditions of PBAC recommendation may limit Australians who could benefit from KALYDECO

The Pharmaceutical Benefits Advisory Committee has again recommended KALYDECO (ivacaftor) for listing on the Pharmaceutical Benefits Scheme (PBS). However, Vertex is deeply disappointed in the conditions the PBAC has attached to the recommendation. These conditions fail to reflect the substantial clinical benefits recognised by the PBAC, and would result in limiting the number of eligible Australians who would be able to receive KALYDECO.

30 Apr 2014

Forest Laboratories enters into definitive agreement to acquire Furiex

Forest Laboratories, Inc. nd Furiex Pharmaceuticals, Inc. today announced that Forest has entered into a definitive agreement to acquire Furiex, a drug development collaboration company, for $95 per share, or approximately $1.1 billion in cash, and up to $30 per share (approximately $360 million in aggregate) in a Contingent Value Right (CVR) that may be payable based on the status of eluxadoline, Furiex's lead product, as a controlled drug following approval.

28 Apr 2014

Bayer's Ciprofloxacin DPI receives FDA orphan drug designation for NCFB treatment

Bayer HealthCare today announced that the U.S. Food and Drug Administration's Office of Orphan Products Development has granted orphan drug designation for its investigational Ciprofloxacin Dry Powder for Inhalation (Ciprofloxacin DPI) for the treatment of non-cystic fibrosis bronchiectasis (NCFB).

23 Apr 2014

Researcher explores public perceptions related to newborn screening programs

While 94 per cent of Canadians surveyed said they would participate in public health programs that screen newborns for a specific number of genetic conditions, only 80 per cent said they would be willing to participate in screening that would sequence their newborns' genomes.

22 Apr 2014

FTC requests additional information relating to Actavis’ pending acquisition of Forest Laboratories

Actavis plc and Forest Laboratories, Inc. today announced that they have each received a request for additional information from the Federal Trade Commission ("FTC") in connection with Actavis' pending acquisition of Forest.

18 Apr 2014

OPKO Health enters into definitive agreement to acquire Inspiro Medical

OPKO Health, Inc. has entered into a definitive agreement to acquire Inspiro Medical Ltd., an Israeli medical device company developing a new platform to deliver small molecule drugs such as corticosteroids and beta agonists or larger molecules to treat respiratory diseases. Inspiro's Inspiromatic is a "smart" easy-to-use dry powder inhaler with several advantages over existing devices.

17 Apr 2014

Alcresta signs agreement with CFFT to support nutritional status of people with cystic fibrosis

Alcresta, a leading medical nutrition company developing innovative enzyme-based products for individuals with unique nutritional needs battling acute conditions or chronic diseases, today announced the company has signed an agreement with Cystic Fibrosis Foundation Therapeutics (CFFT) to accelerate the development of Alcresta's enzyme-based point-of-care products to support the nutritional status of people with cystic fibrosis (CF).

17 Apr 2014

Doc receives grant for helping young adults to move from child-centered to adult-oriented health care system

Oscar Taube, M.D., director of the Pediatric Outpatient department and the coordinator of Adolescent Medicine at the Herman & Walter Samuleson Children's Hospital at Sinai, has been awarded a grant from the American Academy of Pediatrics to provide adolescents, young adults and their family members with the tools to make a smooth transition from pediatric medical care to adult medical care.

10 Apr 2014

Forest Laboratories, Almirall receive feedback from FDA regarding fixed dose combination of aclidinium/formoterol

Forest Laboratories, Inc. and Almirall, S.A. today announced that they have recently received feedback from the U.S. Food and Drug Administration (FDA) regarding the fixed dose combination of aclidinium and formoterol.

9 Apr 2014

Study shows critical role of immune system in protecting against viruses and cancer cells

A team of researchers at the IRCM, led by Andr- Veillette, MD, explains how our immune system kills abnormal blood cells. Their discovery, recently published in the Journal of Experimental Medicine, could eventually lead to new treatment avenues for leukemia, lymphoma and certain types of infectious viral diseases.

9 Apr 2014

Thomson Reuters, Children's Tumor Foundation partner to create neurofibromatosis pathway maps

The Intellectual Property & Science business of Thomson Reuters, the world's leading provider of intelligent information for businesses and professionals, today announced an expansion of its partnership with the Children's Tumor Foundation to create unique neurofibromatosis (NF) pathway maps aimed at significantly increasing the understanding of the disease and its underlying mechanisms.

4 Apr 2014

Forest Laboratories, Richter report positive results from cariprazine Phase IIb trial for bipolar depression

Forest Laboratories, Inc. and Gedeon Richter Plc. today announced positive topline results from a Phase IIb trial evaluating the efficacy and safety of the investigational antipsychotic cariprazine in patients with bipolar depression.

31 Mar 2014

Research findings help explain rare genetic disorder that causes immunodeficiency syndrome

IRCM researchers led by Javier M. Di Noia, PhD, uncovered a new function of AID, a crucial enzyme for the immune response. The discovery, recently published by the scientific journal Proceedings of the National Academy of Sciences, helps explain a rare genetic disorder that causes an immunodeficiency syndrome.

28 Mar 2014

Johns Hopkins researchers identify protein that regulates the body's immune response to CMV

Infectious disease specialists at the Johns Hopkins Children's Center have identified a protein that regulates the body's immune response to cytomegalovirus (CMV), a common pathogen that causes lifelong infections and can lead to devastating illness in newborns and those with weakened immune systems.

27 Mar 2014

Cystic Fibrosis News and Research

Latest Cystic Fibrosis News and Research

EMA's CHMP gives positive opinion for PTC Therapeutics' Translarna

Aradigm receives QIDP designation for its inhaled antibiotic candidate, Pulmaquin

Study shows technology improves transition into adulthood for adolescents with chronic disease

Aradigm reports total revenue of $6.6 million for Q1 2014

Trends, opportunities and threats in the life science industry: an interview with Bob Coughlin, CEO, MassBio

FDA approves Parion’s investigational new drug application for treatment of dry eye disease

Vertex: Conditions of PBAC recommendation may limit Australians who could benefit from KALYDECO

Forest Laboratories enters into definitive agreement to acquire Furiex

Bayer's Ciprofloxacin DPI receives FDA orphan drug designation for NCFB treatment

Researcher explores public perceptions related to newborn screening programs

FTC requests additional information relating to Actavis’ pending acquisition of Forest Laboratories

OPKO Health enters into definitive agreement to acquire Inspiro Medical

Alcresta signs agreement with CFFT to support nutritional status of people with cystic fibrosis

Doc receives grant for helping young adults to move from child-centered to adult-oriented health care system

Forest Laboratories, Almirall receive feedback from FDA regarding fixed dose combination of aclidinium/formoterol

Study shows critical role of immune system in protecting against viruses and cancer cells

Thomson Reuters, Children's Tumor Foundation partner to create neurofibromatosis pathway maps

Forest Laboratories, Richter report positive results from cariprazine Phase IIb trial for bipolar depression

Research findings help explain rare genetic disorder that causes immunodeficiency syndrome

Johns Hopkins researchers identify protein that regulates the body's immune response to CMV

Digital pathology and the PathoVerse: A conversation with Dr. Singh

Bioprocess Efficiency with Cell Density Monitoring

Exploring electron microscopy for plant and microbial imaging at the John Innes Centre

Cystic Fibrosis News and Research

Latest Cystic Fibrosis News and Research

Digital pathology and the PathoVerse: A conversation with Dr. Singh

Bioprocess Efficiency with Cell Density Monitoring

Exploring electron microscopy for plant and microbial imaging at the John Innes Centre

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