Sentences with phrase «rare pediatric diseases»
We're also working with F. Sessions Cole (St. Louis Children's Hospital) on the genomics of rare pediatric diseases.
http://massgenomics.org/
The rare pediatric disease priority review voucher program under section 529 of the Federal Food, Drug, and Cosmetic Act (21 U.S.C. 360ff).
Not exact matches
As a new generation of gene therapy clinical trials shows promise to cure or halt the progression of several rare diseases, the time has come to explore ways to pay for the cutting edge treatments, a pediatric hematologist - oncologist from Dana - Farber / Boston Children's Cancer and Blood Disorders Center argues in a commentary published by the journal Science.
My recent post on rare diseases and pediatric pain clearly resonated with a number of people, prompting my immersion in the medical literature and speaking with some experts and patients about these topics and about the difficulties patients with atypical symptoms -LSB-...]
«While pediatric cancer survivor rates are generally high, there are those children — like Brady — who unfortunately lose their fight due to rare forms of the disease.
Our first liver therapeutic development program is for methylmalonic acidemia (MMA), a serious and often life - threatening rare liver disease; it is primarily a pediatric disease with onset in early infancy.
Although pediatric cancer is rare, it's the leading cause of disease - related death among children who survive past infancy (in the Western world).
Chicago, Ill., July 20, 2016 — AveXis, Inc., a clinical - stage gene therapy company developing treatments for patients suffering from rare and life - threatening neurological genetic diseases, today announced the U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy Designation for AVXS - 101, the company's lead development candidate for the treatment of spinal muscular atrophy (SMA) Type 1 in pediatric patients.
193/6: 30 The hunt for rare disease diagnosis: Utilization of social media, model organisms, and pathway analysis in pediatric exome sequencing.