Life Cycle of Orphan Drug Development and Commercialization Conference

•October 31, 2012 • Leave a Comment

FOR IMMEDIATE RELEASE: (Click here for .pdf of Press Release)

For further information, please contact:

Robin Yegelwel
Marketing & PR Coordinator
marcus evans
455 N. Cityfront Plaza Dr., 9th Floor
Chicago, IL 60611
(312) 540-3000 ext. 6483

Alnylam Pharmaceuticals, BioMarin Pharmaceutical Inc., and Cytonet Set to Speak on Conducting Effective Orphan Drug Research

Barbara Wuebbels, R.N., M.S., Oved Amitay, and Rod Monroy, Ph.D. set to speak at the Premier marcus evans Life Cycle of Orphan Drug Development & Commercialization Conference

Boston, MA – September 19, 2012 – marcus evans, a world leading provider of strategic conferences is pleased to introduce the Life Cycle of Orphan Drug Development & Commercialization Conference, January 16-18, 2013 in Boston, Mass.

The orphan drug and rare disease industry is one of the hottest topics in modern day medicine. “A unique set of patients with rare diseases and severe unmet medical needs exists; it is essential to have people and companies dedicated to develop therapeutic agents to improve the quality of life for these patients,” said Rod Monroy, Ph.D., Senior Director, North American Clinical Operations at Cytonet. With blockbuster drugs coming off patent, there is a great need for pharmaceutical companies to diversify their portfolios by exploring niche markets. The increasing presence of high-profile manufacturers in the orphan drug industry provides these companies with this opportunity.

Orphan drug development has several advantages including faster clinical trials, better odds for approval with an 82% success rate, attractive profitability, given the smaller patient populations, and ongoing revenue growth.

While the orphan drug market offers an attractive future for the pharmaceutical industry, it is not without challenges, which include clinical trial end points and lack of biomarkers and difficulty identifying patients. “Finding key opinion leaders and funding the clinical trial are two of the major challenges in orphan drug development,” said Barbara Wuebbels, Associate Director, Patient Advocacy & Investigator Relations at BioMarin Pharmaceutical Inc.

Growing regulatory issues and demands, a changing reimbursement environment, and outdated distribution models add uncertainty and increase the financial risks. “As we continue to drive scientific innovation to the benefit of patients we need to work with other stakeholders on evolving the commercial paradigms to ensure sustainable long-term success,” said Oved Amitay, Vice President and Head of Commercial at Alnylam Pharmaceuticals.

The Life Cycle of Orphan Drug Development & Commercialization Conference will focus on the current landscape of rare disease and orphan drug development, different forms of structural based drug designs, and specifically designed clinical trials. Through these clinical trials, and by having connected to the right patients to participate, will increase productivity and expedite orphan drug approval and commercialization of the final product.

For more information regarding this conference and to register, please visit http://www.marcusevansch.com/LCOD2012_PR or contact Robin Yegelwel, Marketing & PR Coordinator, at (312) 540-3000 ext. 6483 or RobinY@marcusevansch.com.

About marcus evans

marcus evans conferences annually produce over 2,000 high quality events designed to provide key strategic business information, best practice and networking opportunities for senior industry decision-makers. Our global reach is utilized to attract over 30,000 speakers annually; ensuring niche focused subject matter presented directly by practitioners and a diversity of information to assist our clients in adopting best practice in all business disciplines.

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Hope for Harlow

•March 29, 2012 • Leave a Comment

MarbleRoad recently learned of a family who desperately needs your help ::

Their 6-month old daughter is in need of a liver transplant. Her parents are doing everything they can to make ends meet, but without benefits, times are tough. If you want to add some good karma to the world, this is an easy way to help a very deserving family!

Currently, Harlow is at The Children’s Hospital of Philadelphia, and she is bleeding from her stomach (and possibly also her brain). This young child’s liver is failing and she needs a transplant. The hospital is looking for an O+ blood type with petite build for a possible donor match. They are also looking for folks who might be able to donate blood. For anybody with questions, or who things they may be able to help, please contact the Living Donor Coordinator, Linda Wood, at (215) 615-0564. You might also want to consider a donation to help support the family; donate here –  http://hopeforharlow.chipin.com/h0pe-for-harlow.

Harlow was diagnosed with Alpha-1-Antitrypsin Deficiency :: 

Alpha-1 Antitrypsin Deficiency (Alpha-1) is a genetic condition that is passed on by parents to their children through their genes, and can cause serious liver disease in children and liver and/or lung disease in adults. Alpha-1 occurs when there is a severe lack of a protein in the blood called alpha-1 antitrypsin (AAT) that is mainly produced by the liver. The main function of AAT is to protect the lungs from inlammation caused by infection and inhaled irritants such as tobacco smoke. The low level of AAT in the blood occurs because the AAT is abnormal and cannot be released from the liver at the normal rate. This leads to a build up of abnormal AAT in the liver that can cause liver disease.

You might recall, we referred to the average cost of treatment for alpha-1 in our post “Anti-ACA Litigation, Extenders Act and Rare Disease Day 2011” — $150,000 per year!

From the Johns Hopkins Childrens Center ::

There is no cure for A1AT deficiency. Therapy involves controlling symptoms and attempting to prevent the development of complications. It is very important for the affected person and those around him/her never to smoke in order to protect their lungs. Vaccinations to protect against viruses that can further damage the liver, such as Hepatitis A and Hepatitis B, should be given. It is difficult to predict how severely a liver will be affected. Liver function tests are followed over time. Some patients may have minimal liver disease during their entire lifetime; however a small number will eventually require a liver transplant.

For more information about the disorder visit the Genetic and Rare Diseases Information Center (GARD) on the website for the NIH Office of Rare Diseases Research; or contact the following organizations:

Alpha-1 Advocacy Alliance
PO Box 202
Wolftown, VA 22748
Phone #: 540-948-6777
800 #: 866-367-2122
e-mail: webmaster@alpha1advocacy.org
Home page: http://www.alpha1advocacy.org

Alpha-1 Association
2937 SW 27 Avenue, Suite 106
Alpha-1 Genetic Counselor: 1-800-785-3177
Miami, FL 33133
Phone #: 305-648-0088
800 #: 800-521-3025
e-mail: info@alpha1.org
Home page: http://www.alpha1.org

Alpha-1 Foundation
2937 SW 27th Avenue, Suite 302
Miami, FL 33133 USA
Phone #: 305-567-9888
800 #: 877-228-7321
e-mail: info@alphaone.org
Home page: http://www.alphaone.org

Alpha-1 Research Registry
c/o Medical University of South Carolina
96 Jonathan Lucas St., Suite 812-CSB, MSC 630
Charleston, SC 29425-6300 USA
Phone #: 843-792-0260
800 #: 877-886-2383
e-mail: alphaone@musc.edu
Home page: http://www.alphaoneregistry.org