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Health, science and genetics reporters invited to register now for ACMG annual genetics meeting

Health, science and genetics reporters are invited to register now for the American College of Medical Genetics and Genomics (ACMG) Annual Clinical Genetics Meeting, March 19-23, 2013 at the Phoenix Convention Center.

16 Jan 2013

Longer looks: A key Hagel mission is to cut military health costs; Medical lessons from a mother's death; How they price - and how we pay for -a $300,000 medicine

As David Brooks and Paul Wolfowitz and many others have shown, it's easy to frame our messy fiscal conundrum in these simple terms of guns vs. butter, or rather, guns vs grandma's buttery-yellow, super-pricey heart medication.

11 Jan 2013

Pfizer, Repligen sign licensing deal to advance spinal muscular atrophy program

Repligen Corporation announced today that it has entered into an exclusive worldwide licensing agreement with Pfizer Inc. to advance Repligen's spinal muscular atrophy (SMA) program, originally in-licensed from Families of SMA (FSMA).

3 Jan 2013

Soligenix's OrbeShield granted FDA oral BDP orphan drug designation

Soligenix, Inc., a development stage biopharmaceutical company, announced today that the Office of Orphan Products Development of the US Food and Drug Administration (FDA) has granted orphan drug designation to OrbeShield (oral beclomethasone 17,21-dipropionate or oral BDP) for the prevention of death following a potentially lethal dose of total body irradiation during or after a radiation disaster.

2 Jan 2013

NORD remembers actor Jack Klugman for support to the Orphan Drug Act

Jack Klugman touched the lives of millions of Americans through his support for what ultimately became the Orphan Drug Act. By portraying on his Quincy, M.E. television show the plight of people with rare diseases with no treatment, Mr. Klugman focused a spotlight on the need for incentives to encourage the development of therapies for small patient populations. He did this in partnership with patients and patient advocates who had been unable to gain national attention until he lent his support.

25 Dec 2012

Molecule imitating a virus may trigger inappropriate immune response

Why would our immune system turn against our own cells? This is the question that the combined Inserm/CNRS/ Pierre and Marie Curie University/Association Institut de Myologie have strived to answer in their "Therapies for diseases of striated muscle", concentrating in particular on the auto-immune disease known as myasthenia gravis.

21 Dec 2012

Iclusig gets FDA approval for two rare blood and bone marrow diseases

The U.S. Food and Drug Administration today approved Iclusig (ponatinib) to treat adults with chronic myeloid leukemia and Philadelphia chromosome positive acute lymphoblastic leukemia (Ph+ ALL), two rare blood and bone marrow diseases.

15 Dec 2012

GSK receives FDA approval for raxibacumab to treat inhalational anthrax

The U.S. Food and Drug Administration today approved raxibacumab injection to treat inhalational anthrax, a form of the infectious disease caused by breathing in the spores of the bacterium Bacillus anthracis. Raxibacumab also is approved to prevent inhalational anthrax when alternative therapies are not available or not appropriate.

14 Dec 2012

FDA grants Orphan Drug designation to Milo Biotechnology's AAV1-FS344 inhibitor

Milo Biotechnology today announced its AAV1-FS344 has been granted Orphan Drug designation from the FDA's Office of Orphan Products Development for treatment of Becker and Duchenne muscular dystrophy. AAV1-FS344 is a gene therapy-delivered myostatin inhibitor that increases muscle strength.

12 Dec 2012

Boehringer Ingelheim to commence volasertib plus LDAC Phase III study for AML

New positive Phase II results from an interim analysis of the randomised Phase I/II study involving the company's investigational haematology/oncology compound volasertib in newly diagnosed patients with acute myeloid leukemia (AML) considered ineligible for intensive remission induction therapy were presented at the 54th American Society of Hematology (ASH) annual meeting in Atlanta, USA.

10 Dec 2012

Scientists identify underlying genetic cause for rare motor neurone disorder

Scientists have identified an underlying genetic cause for a rare disorder of motor neurones, and believe this may help find causes of other related diseases.

7 Dec 2012

New genetic study defines biology behind brain developmental disorder in children

This study describes how a combination of sequencing and mouse models were used to identify the gene responsible for a brain developmental disorder seen in four patients.

1 Dec 2012

Novartis to highlight key data from extensive oncology portfolio at SABCS and ASH meeting

Novartis will highlight more than 140 presentations on key data from its extensive oncology portfolio at the leading year-end scientific meetings devoted to hematology and breast cancer, demonstrating continued innovation in research and development efforts to advance the care of patients with cancer and rare diseases.

30 Nov 2012

UBE3B gene linked to brain development disorder in children

Researchers have defined the gene responsible for a rare developmental disorder in children. The team showed that rare variation in a gene involved in brain development causes the disorder. This is the first time that this gene, UBE3B, has been linked to a disease.

30 Nov 2012

AstraZeneca receives FDA approval for Cometriq to treat medullary thyroid cancer

The U.S. Food and Drug Administration today approved Cometriq (cabozantinib) to treat medullary thyroid cancer that has spread to other parts of the body (metastasized).

29 Nov 2012

ArmaGen closes $17M Series A financing

ArmaGen Technologies, Inc. today announced the closing of a $17 million Series A financing to support the development of brain-penetrating recombinant protein therapeutics.

29 Nov 2012

Lymphopenia driven diseases and the orphan drug industry: an interview with Damian Marron, CEO of Cytheris

Lymphopenia is when you lack the T cells of your immune system. T cells are very important for controlling infection. Probably the most known cause of lymphopenia is HIV.

23 Nov 2012

Researchers discover new gene for susceptibility to Alzheimer's disease

A large-scale international study involving French researchers from the Inserm-Institut Pasteur Lille-Universit- Lille Nord de France "Public health and molecular epidemiology of ageing-related diseases" joint research unit led by Philippe Amouyel, has just discovered a gene for susceptibility to a rare disease that causes susceptibility to a common one, Alzheimer's disease, providing evidence of the heterogeneous aetiology of Alzheimer's disease.

20 Nov 2012

CFFT announces major expansion of research collaboration with Pfizer

Cystic Fibrosis Foundation Therapeutics Inc. (CFFT), the nonprofit drug discovery and development affiliate of the Cystic Fibrosis Foundation, today announced a major expansion of its research collaboration with Pfizer Inc. designed to discover new drugs to treat people with the most common mutation of CF, Delta F508.

20 Nov 2012