Rare Disease Report


Researchers report positive results for phase 1 trial of ivosidenib in patients with IDH1+ acute myeloid leukemia.
Results of a recent study demonstrate that treatment at a specialized cancer center is associated with better leukemia-specific survival in children, adolescents, and young adults. 
A new phase 3 clinical trial boasts highest survival rates of these T-cell malignancies.
First-in-man phase 1 trial meets its primary endpoints by demonstrating safety and immunogenicity of a mutation-specific IDH1R132H peptide vaccine.
To date, OPTIMISMM is the only phase 3 study in early RRMM to report a significantly and clinically meaningful PFS improvement in LEN-exposed patients.
Prospective genomics can inform subsequent clinical trial analysis aiming at matching outcome with tumor genotyping.
Saby George, MD, FACP, and Dena Battle, president of nonprofit patient advocacy group, KCCure, discussapproval of Opdivo (nivolumab) and Yervoy (ipilimumab) therpay combination for kidney cancer. 
The US FDA has granted Astellas Pharma Inc priority review for gilteritinib for refractory acute myeloid leukemia (AML) with a FLT3 mutation as detected by an FDA-approved test. 
In an article recently published by the Institute of Cancer Research, genetic testing was indicated to result in life-extending treatment for incurable pediatric brain tumors. 
Yesterday, UroGen Pharma Ltd released positive data from its interim analysis of the ongoing pivotal Phase 3 OLYMPUS clinical trial of UGN-101 (MitoGel) for the non-surgical treatment of low-grade upper tract urothelial cancer (UTUC).
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