John Hornsby Sr., a native of Kentucky, suffered for six months with what he thought was a hernia. John was wrong. He had incurable Non-Hodgkin’s follicular lymphoma, and surgery…
Those with multiple myeloma within the European Union will soon have access to a new therapeutic option. An article for Cancer Network shares that the European Commission (EC) approved…
According to a story from Neuro Central, pharmaceutical company Merck has provided an update on its findings from its phase 3 clinical trial KEYNOTE-412. This clinical trial was evaluating pembrolizumab…
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…
Written By Ashely Winslow, PhD, President & Chief Scientific Officer of Odylia Therapeutics One of the biggest issues impacting many rare disease communities is the availability of approved treatments. While…
According to a story from Markets Insider, the drug company Kazia Therapeutics Limited recently announced that its investigational therapy paxalisib has been awarded Rare Pediatric Disease designation from the US…
For months, those within the rare disease community have been watching to see whether Upstaza (eladocagene exuparvovec) would be approved for use within the European Union. In fact, in…
A Phase 2 clinical trial which sought to evaluate the safety, efficacy, and tolerability of NP-120 (ifenprodil) for idiopathic pulmonary fibrosis (IPF) had some surprising results. Initially, the study…
Dr. Cecilia Chung and associates at the Vanderbilt University’s Medical Center conducted a study published recently in MedPage Today. Results showed that patients who had inflammatory disorders, as well…
According to a recent article, the parents of a boy with epilepsy were told they would need to prepare for him to have end-of-life care after they were unable…
In 2020, Anthony Di Laura and Jackie Cucullo were over-the-moon. The married couple had just learned that Jackie was pregnant with their first child. Anthony and Jackie were thrilled…
Scientists have known for years that Wiskott-Aldrich syndrome (WAS) is caused by a gene on the X chromosome. The gene (the WASP gene) can be found throughout the immune…
In a recent news release, clinical-stage biotechnology company Amolyt Pharma ("Amolyt") shared that positive data was available from a proof-of-concept study evaluating AZP-3601 for hypoparathyroidism. Within this study, researchers sought…
Takeda and its collaborator, Arrowhead Pharmaceuticals, recently announced results from AROAAT-2002 of the investigational drug fazirsiran. The drug is an RNA interference therapeutic developed to lessen alpha-1 antitirypsin protein…
Have you ever heard of Orphan Drug designation? In the United States, this designation is granted by the U.S. Food and Drug Administration (FDA) to drugs or biologics intended…
As time runs out, volunteer Dominic Gillen is running even harder. In an interview with KETV, he admits that they are not yet where they should be in order…
According to a story from Medpage Today, patients using the drug azathioprine who carried a certain genotype were three times more likely to need to either cease treatment or reduce…
Recently, the International Society on Thrombosis and Haemostasis (ISTH) held its 2022 Congress from July 9th to 13th. During the conference, researchers and other stakeholders discussed research, care, and…
CBC News Canada carried the story of a 23-year-old patient, Morgan Buyaki of Saskatchewan, Canada, who was diagnosed eighteen months ago with eosinophilic granulomatosis with polyangiitis (EGPA). Morgan’s mother…
Earlier this year, the European Renal Association-European Dialysis and Transplant Association (ERA-EDTA) held its International Congress. During the Congress, researchers discussed the latest in research and scientific innovation within…
Early diagnosis and treatment is crucial for patients with primary hyperoxaluria type 1 (PH1). Unfortunately, due to a high prevalence of misdiagnosis, as well as poor therapeutic access in…
According to a story from Globe Newswire, the rare disease company Albireo Pharma, Inc. has recently announced the release of new data from its phase 3 trials. These trials were…
Currently, ZYNLONTA (loncastuximab tesirine-lpyl) is approved for the treatment of individuals with relapsed or refractory (R/R) diffuse large B-cell lymphoma (DLBCL) for which two prior therapies had not worked.…
The most common form of leukemia in Western countries is chronic lymphocytic leukemia (CLL). The disease accounts for almost thirty percent of known leukemia cases. Approximately 30,000 Europeans were…
According to a story from AJMC, there are a number of factors in play when determining the path forward for the treatment of advanced melanoma. For this story, Dr. Omid…
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