Rare Disease Report

Oncology

Investigators have discovered an important link between recessive dystrophic epidermolysis bullosa and squamous cell carcinoma, suggesting a new approach to treatment. 
The FDA has granted an orphan drug designation to OBI Pharma, Inc.’s OBI-3424 for the treatment of acute lymphoblastic leukemia.
The FDA has granted an orphan drug designation to Cytori Therapeutics’ ATI-1123 chemotherapy drug for the treatment of small cell lung cancer.
Stay up-to-date on the latest rare disease news by checking out our top 5 articles of the week.
An investigative team from St. Jude Children’s Research Hospital have identified weaknesses to target in rhabdomyosarcoma, leading to a promising precision medicine treatment that has already hit clinical trials.
E. Anders Kolb, MD, discusses the exciting possibilities of using genomic technologies to stratify pediatric cancer patients.
The FDA has granted a priority review for an anti-PD-1 therapy for the treatment of adults and pediatric patients with recurrent locally advanced or metastatic Merkel cell carcinoma.
Stay up-to-date on the latest rare disease news by checking out our top 5 articles of the week.
Investigators from McGill University have discovered a new link connecting immune cells and the development of gastric tumors in those with Peutz-Jeghers Syndrome.
 
The FDA has granted a fast track designation to CX-01 for the treatment of patients over 60 years of age receiving induction therapy for newly-diagnosed AML.
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