Julie Raskin

The FDA has granted orphan drug status to Biodel’s stable glucagon for congenital hyperinsulinism patients. This represents one more important milestone in the development of this drug for HI patients. This FDA designation creates a range of financial incentives for the further development of the drug. Earlier this year, Biodel had received orphan drug designation … Read more

Congenital Hyperinsulinism International (CHI) is happy to announce that Dr. Diva de Leon of the Children’s Hospital of Philadelphia has just been awarded a $50,000 grant to study “The Effect of GLP-1 Receptor Antagonism on Protein-Induced Hypoglycemia in KATP HI.” The study will examine the effect of exendin-(9-39) — a potent glucagon-like peptide (GLP-1) receptor … Read more

Tonight the artwork of Kivrin Hopkins, a nine year old girl with congenital hyperinsulinism, will be displayed at the Rare Voices Gala in Washington DC, honoring members of congress, congressional staff and patient advocates who have raised the profile of rare diseases. Kivrin’s artwork is a 2012 Grand Prize winner of the Rare Artist Contest … Read more

Yesterday I wrote about the very positive near future projections for investments into rare disease drug research and development. We need all of you to take action now to insure that this bright future isn’t threatened by “sequestration,” the automatic budget cuts that are scheduled to take place on January 1, 2013 if action isn’t … Read more

At a conference I attended last month in Washington DC, I learned that investors, pharmaceutical companies and researchers see rare disease treatments as a potential “next frontier” in the world of drug and device development. We’re living in a time when the blockbuster meds designed for more common ailments may be becoming less profitable while … Read more