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Lumena Pharmaceuticals initiates LUM001 clinical program in children with ALGS

Lumena Pharmaceuticals, a company developing oral therapeutics for rare liver diseases, today announced the initiation of a global clinical program to evaluate its drug candidate LUM001 in children with Alagille syndrome. The first patient has been dosed in the IMAGO Phase II study being conducted in the U.K., and enrollment of pediatric patients is expected to begin later this year in the ITCH Phase II study in the U.S.

26 Sep 2013

FDA awards grants to boost development, availability of medical devices for children

The U.S. Food and Drug Administration today announced it has awarded seven grants totaling more than $3.5 million to various pediatric device consortia to boost the development and availability of medical devices for children.

26 Sep 2013

FDA grants orphan drug designation to Soligenix’s SGX94 for treatment of Acute Radiation Syndrome

Soligenix, Inc., a clinical stage biopharmaceutical company focused on developing products to treat inflammatory diseases and biodefense countermeasures where there remains an unmet medical need, announced today that the Office of Orphan Products Development of the US Food and Drug Administration (FDA) has granted orphan drug designation to the active ingredient SGX94 for the treatment of acute radiation syndrome (ARS).

16 Sep 2013

Four new pre-clinical drug development projects at NIH develop treatment for rare disease

Four new pre-clinical drug development projects at the National Institutes of Health will target a form of blindness and diseases characterized by cardiac problems. The projects were selected for their potential to treat specific rare diseases and to help scientists uncover new information that can be shared with other researchers.

13 Sep 2013

FDA approves use of abraxane to treat patients with late-stage pancreatic cancer

The U.S. Food and Drug Administration today expanded the approved uses of Abraxane (paclitaxel protein-bound particles for injectable suspension, ablumin-bound) to treat patients with late-stage (metastatic) pancreatic cancer.

7 Sep 2013

Argininosuccinic aciduria (ASA) and gene therapy: an interview with Dr Julien Baruteau, UCL Institute for Women's Health, London

This rare genetic condition can theoretically affect patients at any age but symptoms are more often observed in childhood and even uncommonly during the first days of life.

2 Sep 2013

Researchers discover new gene mutations which cause devastating mitochondrial disorders

Researchers have identified a novel disease gene in which mutations cause rare but devastating genetic diseases known as mitochondrial disorders.

30 Aug 2013

ASO may correct striatal transcriptional abnormalities and improve behavioral problems in HD mice

Findings from postmortem studies of the brains of Huntington's Disease (HD) patients suggest that transcriptional dysregulation may be an early step in the pathogenesis of HD before symptoms appear. Other studies report transcriptional alterations in the brains of some mouse models of HD.

27 Aug 2013

Powerful new option for treating vasculitis

In a study published in the New England Journal of Medicine, a research consortium that includes the University of Alabama at Birmingham (UAB) has shown that a new drug for vasculitis, a potentially life-threatening auto-immune disease which causes inflammation in blood vessels, is as effective as standard therapy over 18 months.

27 Aug 2013

Shire extends existing partnership with Santaris Pharma in rare genetic disease space

Santaris Pharma A/S, a clinical-stage biopharmaceutical company focused on the discovery and development of RNA-targeted therapies, today announced that its long term partner, Shire has extended the existing partnership in the rare genetic disease space.

23 Aug 2013

Oxford Gene Technology releases next generation sequencing survey results

Oxford Gene Technology, provider of innovative genetics research and biomarker solutions to advance molecular medicine, has released the results from a recent survey of researchers using next generation sequencing.

From Oxford Gene Technology 21 Aug 2013

Walmart stores join together to help Cincinnati Children's battle epidermolysis bullosa disease

This year, 32 Tri-State Walmart stores are joining together to help Cincinnati Children's battle a rare, devastating disease: Epidermolysis Bullosa (EB).

21 Aug 2013

FDA grants Orphan Drug Designation to Alnylam ALN-AT3 for treatment of hemophilia B

Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), a leading RNAi therapeutics company, announced today that the U.S. Food & Drug Administration (FDA) has granted an Orphan Drug Designation to ALN-AT3 as a therapeutic for the treatment of hemophilia B.

17 Aug 2013

FDA grants Orphan Drug Designation to ADXS-HPV for treatment of HPV-associated anal cancer

Advaxis, Inc., (OTCQB: ADXS), a leader in developing the next generation of immunotherapies for cancer and infectious diseases, announced that it has been granted Orphan Drug Designation from the U.S. Food and Drug Administration (FDA) Office of Orphan Products Development (OOPD) for ADXS-HPV, its lead drug candidate, for the treatment of human papillomavirus (HPV)-associated anal cancer.

17 Aug 2013

Stillbirth research: an interview with Dr Lucy Higgins, University of Manchester

Stillbirth, the birth of a baby who shows no sign of life after 24 completed weeks of pregnancy, occurs in 1 in every 200 pregnancies in the United Kingdom. That’s roughly 10 bereaved families a day.

From The University of Manchester 12 Aug 2013

Eisai's investigational compound receives orphan drug designation for cutaneous t-cell lymphoma

Eisai Inc. announced today that the U.S. Food and Drug Administration has granted orphan drug designation to its investigational compound (E7777) for cutaneous t-cell lymphoma. E7777 is designed to have an improved purity profile and manufacturing process.

9 Aug 2013

European authorisation of Aegerion Pharmaceuticals' Lojuxta brings hope for HoFH patients

Aegerion Pharmaceuticals, Inc., a biopharmaceutical company dedicated to the development and commercialisation of novel, life-altering therapies for patients with debilitating, often fatal, rare diseases, announced today that the European Commission has authorised Lojuxta (lomitapide) hard capsules as an adjunct to a low-fat diet and other lipid-lowering medicinal products with or without low density lipoprotein apheresis in adult patients with homozygous familial hypercholesterolaemia.

3 Aug 2013

Trace substance in sugar improves muscle regeneration in mouse model with muscular dystrophy

A trace substance in caramelized sugar, when purified and given in appropriate doses, improves muscle regeneration in a mouse model of Duchenne muscular dystrophy. The findings are published today, Aug. 1, in the journal Skeletal Muscle.

2 Aug 2013

Viropharma announces financial results for second quarter of 2013

VIROPHARMA INCORPORATED (Nasdaq: VPHM) today announced financial results for the second quarter of 2013. Net sales were $104 million for the second quarter ended June 30, 2013 as compared to $95 million in the comparative period of 2012.

1 Aug 2013

FDA grants orphan-drug designation for Ligand’s Captisol-enabled Topiramate Injection

Ligand Pharmaceuticals Incorporated announced that the U.S. Food and Drug Administration (FDA) has granted orphan-drug designation for its proprietary Captisol-enabled Topiramate Injection for the treatment of partial onset or primary generalized tonic-clonic seizures in hospitalized epilepsy patients who are unable to take oral topiramate.

26 Jul 2013