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Stem cell-based epilepsy approach may help in search for better medicines to treat seizure disorders

A new stem cell-based approach to studying epilepsy has yielded a surprising discovery about what causes one form of the disease, and may help in the search for better medicines to treat all kinds of seizure disorders.

25 Jul 2013

Verastem's defactinib gets FDA orphan drug designation for treatment of mesothelioma

Verastem, Inc., focused on discovering and developing drugs to treat cancer by the targeted killing of cancer stem cells, announced that lead cancer stem cell inhibitor, VS-6063 (defactinib), has received orphan drug designation from the U.S. Food and Drug Administration for use in the treatment of mesothelioma, a rare form of lung cancer.

24 Jul 2013

Child cancer survival: an interview with Prof Kathy Pritchard-Jones, University College London

Back in the early 1960s, just before chemotherapy really started to be used, only about 30% of children survived cancer. That was mainly because they had cancers that were amenable to surgery.

24 Jul 2013

Experts to focus on health care challenges at U.S. Conference on Rare Diseases and Orphan Products

Patient advocates, industry leaders, medical researchers, government officials, and investors will join together in Bethesda, MD, on Oct. 7 - 9 for the 3rd annual U.S. Conference on Rare Diseases and Orphan Products.

20 Jul 2013

Viewpoints: Expanding abortion restrictions; Lift the ban on gay blood donors; Medicare's price schedules

What's going on in Wisconsin, North Dakota, South Dakota and Iowa ought to be a wake-up call to Minnesotans who prefer that physicians, not politicians, guide an individual's medical care. Minnesota's neighbors have moved with alarming alacrity to cripple women's access to a safe and legal medical procedure - abortion (7/18).

20 Jul 2013

Absence of Shp-2 enzyme near cartilage cells can lead to multiple benign cartilage tumors in mice

Rhode Island Hospital researchers have found that the absence of the Shp-2 enzyme near specialized cartilage cells can lead to the development of multiple benign cartilage tumors in mice, a model that recapitulates the rare human tumor syndrome metachondromatosis.

20 Jul 2013

FDA grants orphan drug designation to ARMAGEN's AGT-182 for treatment of Hunter syndrome

ARMAGEN announced today that the U.S. Food and Drug Administration has granted orphan drug designation to its lead product AGT-182 for the treatment of mucopolysaccharidosis type II (also known as Hunter syndrome or MPS II).

19 Jul 2013

Ensemble Therapeutics announces initiation of drug discovery collaboration with Alexion subsidiary

Ensemble Therapeutics, a biotechnology company developing Ensemblins, a novel class of small molecule therapeutics with the potential to modulate targets typically addressed by biologics, today announced the initiation of a drug discovery collaboration with a subsidiary of Alexion Pharmaceuticals, Inc., the global biopharmaceutical company focused on developing and commercializing life-transforming therapies for patients with life-threatening, ultra-rare disorders.

18 Jul 2013

Study identifies new therapeutic targets for Huntington's disease

A scientific study describes that GluN3A subunits of NMDA receptors that interact with glutamate —the main exciting neurotransmitter in the nervous system— are therapeutic targets against Huntington's disease.

16 Jul 2013

Alize Pharma initiates AZP-531 Phase I clinical trial for treatment of type 2 diabetes

Alize Pharma, a company specialized in the development of drugs for the treatment of metabolic diseases and rare diseases, announces today the launch of the first Phase I clinical trial for AZP-531, its unacylated ghrelin analog, in type 2 diabetes.

16 Jul 2013

Action Medical Research funds three new Research Training Fellowships

Action Medical Research, the UK-wide charity funding vital research to help sick and disabled babies and children, has today announced it is funding three new Research Training Fellowships worth more than £470,000.

9 Jul 2013

Biogen, Sobi present new data supporting clinical, safety profile of ELOCTATE for hemophilia A

Biogen Idec nd Swedish Orphan Biovitrum AB (publ) presented new data that support the clinical and safety profile of their long-lasting recombinant factor VIII candidate ELOCTATE for hemophilia A.

4 Jul 2013

Biogen, Sobi present new findings from Phase 3 trial of ALPROLIX at XXIV ISTH Congress

Biogen Idec and Swedish Orphan Biovitrum AB (publ) today presented new findings for their long-lasting recombinant factor IX candidate ALPROLIX for hemophilia B at the XXIV International Society on Thrombosis and Haemostasis Congress in Amsterdam, The Netherlands.

3 Jul 2013

Study: More people are being affected by drug-induced liver injury than ever before

More people are being affected by drug-induced liver injury (DILI) than ever before, according to a new study in Gastroenterology, the official journal of the American Gastroenterological Association.

27 Jun 2013

Advaxis seeks ADXS-HPV Orphan Drug Designation for treatment of HPV-associated anal cancer

Advaxis, Inc., a leader in developing the next generation of immunotherapies for cancer and infectious diseases, announced that it has submitted an Application for Orphan Drug Designation with the U.S. Food and Drug Administration Office of Orphan Products Development for ADXS-HPV, its lead drug candidate, for the treatment of human papillomavirus-associated anal cancer.

25 Jun 2013

NIH awards $12.7M to research groups to explore new treatments for patients in eight disease areas

The National Institutes of Health has awarded $12.7 million to match nine academic research groups with a selection of pharmaceutical industry compounds to explore new treatments for patients in eight disease areas, including Alzheimer's disease, Duchenne muscular dystrophy and schizophrenia.

19 Jun 2013

European Commission amends marketing authorisation for Celgene's REVLIMID

Celgene International Sàrl was today notified that the European Commission has amended the marketing authorisation for REVLIMID. This decision means that REVLIMID is now approved to treat patients with transfusion-dependent anaemia due to low or intermediate-1 risk myelodysplastic syndromes associated with an isolated deletion 5q cytogenetic abnormality when other therapeutic options are insufficient or inadequate.

17 Jun 2013

Researchers identify genetic mutation responsible for MDP Syndrome

National Paracycling Champion Tom Staniford has an extremely rare condition which, until now, has puzzled his doctors. He is unable to store fat under his skin - yet has type 2 diabetes - and suffered hearing loss as a child. Now, thanks to advances in genome sequencing, an international research team led by the University of Exeter Medical School has identified Tom's condition and pinpointed the single genetic mutation that causes it.

17 Jun 2013

NPS Pharmaceuticals president receives Ernst & Young Entrepreneur Award in Life Sciences category

NPS Pharmaceuticals, Inc. (NASDAQ: NPSP), a biopharmaceutical company pioneering and delivering therapies that transform the lives of patients with rare diseases worldwide, today announced that president and chief executive officer Francois Nader, M.D. has received the Ernst & Young Entrepreneur Of The Year® 2013 Award in the Life Sciences category in New Jersey. Dr. Nader was presented with the award at a special gala event on June 13 at the Hyatt Regency New Brunswick, New Jersey.

15 Jun 2013

MemoryShape Breast Implants receive FDA approval

The U.S. Food and Drug Administration today approved the MemoryShape Breast Implant to increase breast size (augmentation) for use in women at least 22 years old and to rebuild breast tissue (reconstruction) in women of any age.

15 Jun 2013

Rare Disease News and Research

Stem cell-based epilepsy approach may help in search for better medicines to treat seizure disorders

Verastem's defactinib gets FDA orphan drug designation for treatment of mesothelioma

Child cancer survival: an interview with Prof Kathy Pritchard-Jones, University College London

Experts to focus on health care challenges at U.S. Conference on Rare Diseases and Orphan Products

Viewpoints: Expanding abortion restrictions; Lift the ban on gay blood donors; Medicare's price schedules

Absence of Shp-2 enzyme near cartilage cells can lead to multiple benign cartilage tumors in mice

FDA grants orphan drug designation to ARMAGEN's AGT-182 for treatment of Hunter syndrome

Ensemble Therapeutics announces initiation of drug discovery collaboration with Alexion subsidiary

Study identifies new therapeutic targets for Huntington's disease

Alize Pharma initiates AZP-531 Phase I clinical trial for treatment of type 2 diabetes

Action Medical Research funds three new Research Training Fellowships

Biogen, Sobi present new data supporting clinical, safety profile of ELOCTATE for hemophilia A

Biogen, Sobi present new findings from Phase 3 trial of ALPROLIX at XXIV ISTH Congress

Study: More people are being affected by drug-induced liver injury than ever before

Advaxis seeks ADXS-HPV Orphan Drug Designation for treatment of HPV-associated anal cancer

NIH awards $12.7M to research groups to explore new treatments for patients in eight disease areas

European Commission amends marketing authorisation for Celgene's REVLIMID

Researchers identify genetic mutation responsible for MDP Syndrome

NPS Pharmaceuticals president receives Ernst & Young Entrepreneur Award in Life Sciences category

MemoryShape Breast Implants receive FDA approval

Designing fluidic architecture for scalable diagnostic platforms

Evaluating new approaches for rare disease genomics

Expanding access to NMR with the Fourier 80 Duo

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