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Positive Subtype Data From the BELIEF Trial Presented at the 12th International Conference on Malignant Lymphoma
Tuesday, June 18, 2013
The belinostat abstract, which will be presented orally at the ICML conference, puts special emphasis to the BELIEF trial’s subtype AITL, which has shown an objective response rate (ORR) of 45.5%. The abstract further concludes that the favorable safety profile observed warrants further “The P
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Native American Myopathy: A Breakthrough in Rare Disease That Only Affects Lumbees
Monday, June 17, 2013
When her son, Gene, was only a few days old, Cassandra Locklear said doctors labeled him with a foreboding prognosis: "failure to thriveThe muscles in his mouth were so weak that he couldn't suck a bottle, Locklear saidDoctors weren't sure what was wrong, and, without the ability to get the nutri
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Rare Disease Hits Young King George Father
Monday, June 17, 2013
Nichole Langa was describing the way a rare disease changed her husband from a vibrant athlete, enrolled in a leadership program with the federal government, to a man who can barely walk or talk and has gauze stuffed in his mouth to absorb excess salivaAt times, she stopped and looked into the dar
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Family Seeks Help for Child With Rare Disease
Monday, June 17, 2013
Janiya suffers from spinocerebellar ataxias. It is a hereditary defect in a certain gene that makes abnormal proteins. The abnormal proteins hamper the ability of nerve cells, primarily in the cerebellum and spinal cord, to function properly and cause them to degenerate over time. As the disease A
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XOMA Initiates Pilot Trial Studying Gevokizumab in Patients With Pyoderma Gangrenosum
Monday, June 17, 2013
XOMA Corporation, a leader in the discovery and development of therapeutic antibodies, today announced it has opened enrollment in a pilot study to determine gevokizumab's potential to treat acute inflammatory pyoderma gangrenosum. Pyoderma gangrenosum (PG) is one of the several rare diseases that
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Amicus Therapeutics to Host Conference Call Today at 8:00 a.m. ET to Discuss Regulatory Strategy for Migalastat HCl Monotherapy for Fabry Disease
Monday, June 17, 2013
Amicus Therapeutics, a biopharmaceutical company at the forefront of therapies for rare and orphan diseases, announced that John F. Crowley, Chairman and Chief Executive Officer, will host a conference call at 8:00 a.m. ET this morning. Mr. Crowley will highlight the regulatory strategy for Intere
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Oral Apremilast Achieves Statistical Significance for Primary and Secondary Endpoints in a Phase II Trial in Patients With Behçet’s Disease
Monday, June 17, 2013
Celgene International Sàrl, a subsidiary of Celgene Corporation presented results from a randomized, placebo-controlled phase II trial in patients with Behçet’s disease (BD) at EULAR, the European Congress of Rheumatology annual meeting in MadridThe Company announced statistical significance f
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Oral Anti-Cancer Therapy REVLIMID® (Lenalidomide) Now Indicated as a Treatment for Patients With Rare Form of Blood Disease
Monday, June 17, 2013
Celgene International Sàrl was notified that the European Commission (EC) 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 (MDS)
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Roche’s Avastin Approved in Japan for Treatment of the Most Aggressive Form of Brain Cancer First Approval of Avastin for the Treatment of Newly Diagnosed Glioblastoma
Monday, June 17, 2013
Roche announced that the Japanese Ministry of Health, Labour and Welfare (MHLW) has approved Avastin (bevacizumab) for the treatment of malignant glioma, including newly diagnosed glioblastoma (GBM) in combination with radiotherapy and temozolomide chemotherapy, and as monotherapy for treatment of
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CTI Announces New Data Presented at EHA 2013 Congress Demonstrating the Safety and Tolerability Profile of Pacritinib in Patients With Myelofibrosis
Monday, June 17, 2013
Cell Therapeutics, Inc. announced results from a pooled analysis of data from completed Phase 1 and 2 studies of pacritinib, an oral JAK2/FLT3 inhibitor, demonstrating the safety and tolerability profile of pacritinib in patients with myelofibrosis. An integrated safety analysis of four completed