Right now, there are a number of therapeutic options for people living with Waldenstrom macroglobulinemia (WM), such as chemotherapy, immunotherapy, radiation, plasmapheresis, stem cell transplantation, and targeted therapy. However, these…
Currently, an estimated 47-50% of global nasopharyngeal carcinoma cases occur in China, with the regions of Guangdong, Guangxi and Fujian being heavily affected. There are a number of treatment…
Could an ocular implant be an effective therapeutic solution for individuals living with noninfectious uveitis? According to an article in HCP Live, the answer seems to be yes. A study…
Also known as Lawrence syndrome, acquired generalized lipodystrophy (AGL) is an ultra-rare condition characterized by the destruction or loss of adipose tissue. Given the small population size, AGL is…
Lia Porcano was born in March 2018—and her parents Rosalyn and Justin could not have been more excited. But when Lia’s newborn screening test came back abnormal, the parents’ concern…
There are many rare or congenital diseases that, without early detection and treatment, can cause serious health issues and complications. For example, early treatment of homocystinuria (HCU) could delay or…
In a news release from commercial-stage biopharmaceutical company Calliditas Therapeutics AB CALT ("Calliditas"), the company shared positive topline results from the Phase 3 NefIgArd study. Within the study, researchers…
Amber Freed has long been a tireless advocate for the rare disease community. After two years of IVF, she became pregnant with her two children: Maxwell and Riley. About four…
When Cheryl Navar decided to have a child, in-vitro fertilization (IVF) seemed like the right move. Navar knew that she had a cancer gene and didn’t want to transfer…
Although the medical field has made significant strides over the years, there is still a lot we don’t know about the body. For example, what can we learn about…
If current estimates are correct, around 10% of people with sarcoidosis within the United States are using off-label TNF-alpha inhibitors such as Remicade (infliximab). But according to an article in…
When Jen and Will Fox remember their son Dalton, they think about his infectious smile, his passion for adventures, and his absolute adoration of baseball. Dalton, who was diagnosed with…
One of the symptoms of scleroderma is the formation of painful ulcers on the fingers. These ulcers are often difficult to manage; they do not respond well to treatment and…
Before you continue reading, make sure to check out Part 1 of Katie's story. True Precocious Puberty: The First Official Diagnosis The endocrinologist first ran a blood test, later followed with a…
Proteinuria refers to excess protein in the urine. In healthy individuals, proteins pass through the kidneys and into the bloodstream. Proteinuria often indicates kidney dysfunction or damage. In fact,…
A primary immunodeficiency (PI), or primary immune deficiency disease, is an inherited immune system disorder caused by gene mutations which prevent the immune system from functioning properly. It can…
There have been rising incidences of gastric cancer on a global scale. As a result, this cancer is now considered the fourth leading oncological cause of death worldwide. Gastric cancer…
A Phase 2a study sought to compare the safety, efficacy, and tolerability of ABBV-3373, as compared to adalimumab, for patients with rheumatoid arthritis (RA). Patients involved in the study had…
The goal of polymyalgia rheumatica treatment is to reduce inflammation and control or manage the other symptoms. As such, the current standard-of-care includes long-term corticosteroid treatment. According to MedPage Today,…
Dr. Muna Naash received her PhD from Baylor College of Medicine, where she studied retinal cell and molecular biology. Throughout her career, Dr. Naash has become known for her work…
Clinical trials can play a crucial role in understanding diseases, as well as identifying potential therapeutic options. This is especially important in newly identified or poorly understood conditions such as…
Prior studies have suggested that metformin, often used as a first-line treatment for people with type 2 diabetes (T2D0, could confer benefits for Rett syndrome. In particular, prior studies found…
In the past, research has found connections between polycystic ovary syndrome (PCOS) and pancreatic cancer. For example, one study suggested that having PCOS could increase pancreatic cancer risk by…
Seizures associated with conditions such as Dravet syndrome and Lennox-Gastaut syndrome (LGS) can be difficult to treat; these seizures may be treatment-averse and may not respond well to current anti-epileptic…
Have you heard of the pulmonary microbiota, which may also be referred to as the lung microbiome? This refers to the complex collection of microorganisms (bacteria, viruses, bacteriophages, fungi) that…
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