For years, there have been few – or no – therapeutic options in Canada for those with fibrodysplasia ossificans progressiva (FOP), a rare genetic connective tissue disorder. However, this has…
Julia Lefelar, a resident of Gaithersburg, MD, was telling a friend about her years of seeing different doctors trying to find the cause of her illness. After a while, her…
Orphan Drug designation is granted to drugs or biologics intending to treat, prevent, or diagnose rare diseases or conditions. For the purpose of the designation, “rare” is considered any condition…
Compassion [kuhm-pash-uhn] noun A feeling of deep sympathy and sorrow for another who is stricken by misfortune, accompanied by a strong desire to alleviate the suffering. Compassion Corner is…
According to a recent article from Hematology Advisor, a study shows patients diagnosed with particular subtypes of diffuse large B-cell lymphoma (DLBCL) respond better to ibrutinib with R-CHOP compared to…
The Critical Path Institute (C-Path), formed in 2005, is a nonprofit, independent (public/private) partnership. The Institute evolved from the FDA’s 2004 White Paper known as the Critical Path Report and…
In a recent news release, enzyme engineering company Codexis, Inc. shared that its product CDX-6512 received both Orphan Drug and Rare Pediatric Disease designations from the FDA. Both designations were…
A recent article published in Physician's Weekly highlighted a study that compared four different treatment regimens for lupus nephritis. The options include low-dose cyclophosphamide (LDCyC), high-dose cyclophosphamide (HDCyC), rituximab, and…
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