Seven years ago, at just 15 years old, Lauren Blake was diagnosed with gastroparesis. This condition makes it difficult for Lauren to keep down her meals or, really, to eat…
Welcome to the Rare Classroom, a new series from Patient Worthy. Rare Classroom is designed for the curious reader who wants to get informed on some of the rarest, most…
The World Health Organization (the WHO) announced World Leprosy Day which this year will be celebrated on January 30th. The theme of the celebration is “United for Dignity.” It…
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…
According to a recent article, a recent study shows evidence that anti-Semaphorin 3B antibodies are pathogenic and therefore patients diagnosed with membranous nephropathy should be monitored after a kidney transplant.…
According to a recent article, a new study has developed a novel index that differentiates eyes with subclinical keratoconus and normal eyes. Keratoconus Keratoconus is a disorder affecting the cornea…
Cannabidiol (CBD) has recently been lauded for its many uses, ranging from stress relief to seizure reductions. Specialty pharmaceuticals company Zynerba Pharmaceuticals (“Zynerba”) developed an experimental cannabidiol gel called Zygel…
Early detection is the key to treating so many types of cancer. Autoimmune hepatitis (AIH), chronic inflammation of the liver, is brought about by an immune system disorder. AIH must…
What do medication, vagus nerve stimulation, and low-carb ketogenic diets all have in common? Currently, these are all therapeutic options for those with Dravet syndrome. However, researchers and doctors believe…
In a news release from January 24, 2022, immuno-pharma company AIM ImmunoTech Inc. ("AIM") shared that positive clinical data was now published. The data, which you can find in Clinical Cancer…
Oscar Warrent, a toddler from Norwich, England, has been through a lot in his two years of life. About three months ago, he was diagnosed with rhabdomyosarcoma, a rare form…
On January 20, 2022, the Rare Disease Legislative Advocates (RDLA) hosted its first monthly webinar for 2022. In this month's program, the speakers provided an overview of the progress that…
An article in PubMed dated October 8, 2021 outlines immune therapies treating myelodysplastic syndrome (MDS) and acute myeloid leukemia (AML) patients. Both cancers are hematologic malignancies that begin in the…
According to a recent article, new evidence shows adding ibrutinib to a patient’s chemotherapy regimen may improve the longevity of younger patients with diffuse large B-cell lymphoma (DLBCL). Diffuse Large…
Glioblastoma is a highly malignant and aggressive brain cancer. With a 5-year survival rate of under 10%, there is an urgent need for newer and more effective diagnostic and treatment…
According to a recent article, the 2021 American Society of Hematology (ASH) Annual Meeting and Exposition displayed the advances that have been made in the management of follicular lymphoma. Follicular…
We have been living in the midst of a pandemic for years now, and with that has come a number of lasting effects. One of these effects, which has been…
According to a recent article, patients diagnosed with minimal change disease are found to have autoantibodies targeting nephrin, which supports previous research. Minimal Change Disease (MCD) Minimal change disease is…
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