FDA Grants Orphan Drug Status For Cyclodextrin Compound To Treat Fatal Genetic Cholesterol Disease
May 16, 2010 by Chris Hempel
Filed under Featured Stories
Drum roll, please! Dr. Caroline Hastings at Children’s Hospital Oakland and Research Center received a call on Friday afternoon from the U.S. Food and Drug Administration regarding the orphan drug submission we made at the end of February. While we have not received the official letter by mail (probably next week), it appears our Orphan […]
Dementia in Children and Teens – When Kids Brains Regress Like The Elderly
March 21, 2010 by Chris Hempel
Filed under Alzheimers, Featured Stories
The Alzheimer’s Society and Alzheimer’s Association have lots of information on caring for people with dementia but no resources on their website for people caring for children, teens and young adults suffering from dementia. There are many kids who have dementia as a result of rare diseases even though most people think dementia strikes the […]
FDAs Orphan Drug Workshop Featured in Wall Street Journal – Push To Cure Rare Diseases
March 10, 2010 by Chris Hempel
Filed under Featured Stories
Below is a story that ran in today’s Wall Street Journal (Page A 3) on the FDA workshop I attended a few week ago at the Keck Graduate Institute in Claremont, CA. We filed an orphan drug application for Cyclodextrin for the treatment of Niemann Pick Type C disease, an ultra rare cholesterol disease that […]
FDA Does Not Approve Actelion’s Zavesca in US – Distressing News For Niemann Pick Type C Community
March 9, 2010 by Chris Hempel
Filed under Featured Stories
Actelion, the makers of Zavesca, today put out a brief statement saying that the U.S. Food and Drug Administration did not approve Zavesca and wants more information from the company on treating Niemann Pick Type C, a rare and dementing neurological disease that afflicts our six year old identical twins, Addi and Cassi. Niemann Pick Type C disease is caused […]
Social Security Disability ‘Compassionate Allowance’ – Only For Those Who are Impoverished?
March 5, 2010 by Chris Hempel
Filed under Health Care Policy
Today I called the Social Security Office in Nevada to find out about the compassionate allowance program for our twins. Last month, the Social Security Administration added 38 more conditions to the already 50 rare diseases and cancers designated for compassionate allowance, which provides expedited review of disability applications from people with severely disabling conditions. […]
