Rare Disease News and Research

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Alexion submits Soliris marketing applications to FDA, EMA for aHUS treatment

Alexion Pharmaceuticals, Inc. announced today that the company has submitted marketing applications to the U.S. Food and Drug Administration and the European Medicines Agency for Soliris as a treatment for patients with atypical Hemolytic Uremic Syndrome.

7 Apr 2011

Sanofi-aventis completes exchange offer for all shares of Genzyme common stock

Sanofi-aventis announced today that it has successfully completed its exchange offer for all outstanding shares of common stock of Genzyme Corporation.

4 Apr 2011

Study confirms D-penicillamine and trientine effective against Wilson disease

Results from the first ever European-wide retrospective analysis presented today at the International Liver CongressTM have shown both D-penicillamine and trientine continue to be effective treatments, providing positive survival rates in patients with Wilson disease free from a liver transplant.

1 Apr 2011

International experts meet to focus on impact of vaccines on global health at a time of public scepticism and complacency

International experts gather in Prague today for a three day meeting focused on the impact of vaccines on public health, at a time of increasing concern that public scepticism and complacency could undermine the positive impact of current and future vaccine campaigns.

31 Mar 2011

Shire's Replagal and VPRIV data on Gaucher and Fabry patients presented at ACMG meeting

Shire plc, the global specialty biopharmaceutical company, presented new data for Replagal in patients with Fabry disease, and VPRIV in patients with type 1 Gaucher disease, at the 2011 American College of Medical Genetics' Annual Clinical Genetics Meeting, held in Vancouver, BC, from March 16-20, 2011.

22 Mar 2011

ViroPharma launches online community for teens and adults with hereditary angioedema

ViroPharma Incorporated today announced the launch of "HAE and Me," an online community that unites teens and adults with hereditary angioedema (HAE) through shared experiences to help them better manage their disease.

21 Mar 2011

Synageva BioPharma closes $25 million private equity financing

Synageva BioPharma Corp., a privately held biopharmaceutical company developing therapeutic products for rare disorders, announced today the closing of a $25 million private equity financing.

21 Mar 2011

Talecris granted EU orphan drug designation for Plasmin (human) to treat acute peripheral arterial occlusion

Talecris Biotherapeutics, Inc. (TLCR) announced today that it has been granted orphan drug designation by the European Commission for the development of Plasmin (human) to treat acute peripheral arterial occlusion (aPAO). Talecris is currently investigating Plasmin in a phase II clinical trial designed to assess its ability to treat aPAO, a condition in which arterial blood flow to the extremities, usually the legs, is blocked by a clot.

4 Mar 2011

Infinity commences IPI-926 Phase 2 clinical trial in rare, life-threatening bone cancer

Infinity Pharmaceuticals, Inc. today announced the initiation of a randomized, double-blind Phase 2 clinical trial to evaluate the safety and efficacy of IPI-926 compared to placebo in patients with metastatic or locally advanced, inoperable chondrosarcoma. The trial is expected to enroll over 100 patients worldwide.

3 Mar 2011

International consortium of researchers genetically characterise Spanish Fanconi anaemia patients

A consortium of thirty-two researchers worldwide, led by Dr Jordi Surrallés, professor of the Department of Genetics and Microbiology at Universitat Autònoma de Barcelona (UAB) and member of the Centre for Biomedical Network Research on Rare Diseases (CIBERER), genetically and clinically characterised almost all Spanish patients suffering from Franconi anaemia, a rare disease affecting one in every 500,000 persons and which is characterised by severe anaemia in children, congenital malformations and a high predisposition to cancer.

3 Mar 2011

Genzyme introduces new global grant program to support LSD patient community

Genzyme Corporation today recognized International Rare Disease Day with the launch of a new program, the Genzyme Patient Advocacy Leadership Awards.

28 Feb 2011

EDMA supports the Rare Disease Day

On the occasion of the 2011 Rare Disease Day, EDMA, the European Diagnostic Manufacturers Association, underlines the crucial role the In Vitro Diagnostic industry can play to diagnose rare diseases.

28 Feb 2011

Synageva supports Fourth International Rare Disease Day

Synageva BioPharma Corp., a privately held biopharmaceutical company that is developing therapeutic products for rare disorders, announced today that the company is supporting the Fourth International Rare Disease Day observance on February 28, 2011.

28 Feb 2011

PNH patients urge government to provide access to life-saving treatment

With so few people affected by rare diseases, how does a group of less than 90 patients across Canada make their voices heardon a life-and-death issue such as access to treatment? Barry Katsof, President of the Canadian Association of PNH Patients, says his group is turning to social media to ask others to take a stand with them in the fight for their lives.

28 Feb 2011

Alexion joins EURORDIS, NORD and medical communities to observe Rare Disease Day 2011

Alexion Pharmaceuticals, Inc. joins the European Organization for Rare Diseases, the National Organization for Rare Disorders and families, governments, companies and medical communities around the world in observing the fourth annual Rare Disease Day today.

28 Feb 2011

Global Genes Project, 46NYC introduce t-shirts to create awareness about lack of drug treatments for rare disease

Wear That You Care. Today is Rare Disease Day and 46NYC is helping raise awareness for an important cause – this time the drug development crisis facing the rare disease patient community worldwide.

28 Feb 2011

Medunik to introduce novel orphan drugs to Canadians with rare diseases

Éric Gervais, Executive Vice-President of Medunik Canada, is proud to announce the Management Team of this recently created company. Medunik Canada is a Quebec pharmaceutical company with 60 employees and one unique mission - bring novel orphan drugs to Canadians with rare diseases.

28 Feb 2011

New study identifies recurrent wound botulism among injection drug users

Botulism is a rare disease and recurrent botulism even more rare. However, in California, recurrent wound botulism among injection drug users has been on the rise and makes up three-quarters of reported cases in the United States.

28 Feb 2011

Enobia completes patient enrollment in ENB-0040 Phase II study for HPP

Enobia Pharma Inc., today announced that it has successfully completed enrollment in its Phase II study of ENB-0040, a bone targeted enzyme replacement therapy being investigated in adolescents and adults with hypophosphatasia, a serious, rare metabolic bone disorder.

25 Feb 2011

Study: Rare genetic mutations may provide clues to improve treatment for schizophrenia

Scientists are eyeing a rare genetic glitch for clues to improved treatments for some people with schizophrenia - even though they found the mutation in only one third of 1 percent of patients.

25 Feb 2011

Rare Disease News and Research

Alexion submits Soliris marketing applications to FDA, EMA for aHUS treatment

Sanofi-aventis completes exchange offer for all shares of Genzyme common stock

Study confirms D-penicillamine and trientine effective against Wilson disease

International experts meet to focus on impact of vaccines on global health at a time of public scepticism and complacency

Shire's Replagal and VPRIV data on Gaucher and Fabry patients presented at ACMG meeting

ViroPharma launches online community for teens and adults with hereditary angioedema

Synageva BioPharma closes $25 million private equity financing

Talecris granted EU orphan drug designation for Plasmin (human) to treat acute peripheral arterial occlusion

Infinity commences IPI-926 Phase 2 clinical trial in rare, life-threatening bone cancer

International consortium of researchers genetically characterise Spanish Fanconi anaemia patients

Genzyme introduces new global grant program to support LSD patient community

EDMA supports the Rare Disease Day

Synageva supports Fourth International Rare Disease Day

PNH patients urge government to provide access to life-saving treatment

Alexion joins EURORDIS, NORD and medical communities to observe Rare Disease Day 2011

Global Genes Project, 46NYC introduce t-shirts to create awareness about lack of drug treatments for rare disease

Medunik to introduce novel orphan drugs to Canadians with rare diseases

New study identifies recurrent wound botulism among injection drug users

Enobia completes patient enrollment in ENB-0040 Phase II study for HPP

Study: Rare genetic mutations may provide clues to improve treatment for schizophrenia

Achieving and maintaining ISO 27001 and BS 10012 certifications

Expanding research and clinical options for children with cancer

Examining how pain could play a direct protective role in the gut

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