The main symptom of Kleine-Levin syndrome – excessive sleepiness (hypersomnolence) – is also a key characteristic of many other sleep disorders, such as narcolepsy. Additionally, there are currently no observed…
Necroptosis, a type of regulated cell death, has been implicated in diseases such as inflammatory bowel disease, as well as neurodegenerative diseases. According to a recent article in News Medical…
Many patients with sickle cell disease (SCD) experience “pain crises,” or periodic episodes of extreme pain. These pain crises occur when sickle-shaped red blood cells block the flow of blood…
In patients with hereditary angioedema (HAE), the C1-inhibitor blood protein does not function properly, allowing bradykinin peptides to build up in the body. These then cause inflammation and fluid leakage;…
According to information from the National Institute of Health, one in nine elderly people in the U.S. will be affected by Alzheimer’s disease (AD). A recent article in the Globe Newswire…
Have you ever heard of acral lentiginous melanoma? This rare form of skin cancer typically develops on nail beds, on the palms of the hands, or soles of the feet.…
Acute myeloid leukemia (AML) is a cancer of the bone marrow and blood. It spreads rapidly. Therefore precise and swift treatment is critical. In a recent interview with Medical Life…
Six years ago, researchers began developing single-domain antibodies (nanobodies) to help identify different species of Ebola; their work can be found published in ACS Infectious Diseases. Initially, the nanobodies were created to…
In the past, there have been a few case reports and other studies which theorized a potential link between eosinophilic conditions, such as eosinophilic esophagitis (EoE), and Celiac disease, a…
The American Association for Cancer Research (AACR) Annual Meeting took place from April 8th until the 13th, with lots of interesting data and innovations presented by some of the leaders…
One of the largest problems that stands in the way of rare disease research is funding. It can be difficult to raise the enormous amounts of money needed to plan,…
In the Phase 3 KEYNOTE-522 clinical trial, researchers found that pembrolizumab was effective in treating patients with triple-negative breast cancer (TNBC). According to Medical XPress, researchers wanted to determine whether…
According to a recent article from WFMZ+, the FDA has recently approved Regeneron's supplemental Biologics License Application (sBLA) for Dupixent, granting it priority review. Dupixent, also known by the generic…
Did you know that an estimated 50% of those with lupus have neuropsychiatric symptoms, or symptoms which impact their mental and psychological health and functioning? For example, some people with…
In the United States, Orphan Drug designation is granted by the FDA to drugs or biologics intended to treat, prevent, or diagnose rare diseases or conditions. “Rare” is defined as…
According to a recent press release, the FDA has recently granted approval to Vijoice - also known under the generic name alpelisib - for the treatment of PIK3CA-related overgrowth spectrum…
A recent study titled "Effect of FT218, a Once-Nightly Sodium Oxybate Formulation, on Disrupted Nighttime Sleep in Patients With Narcolepsy: Results From the Randomized Phase 3 REST-ON Trial," was published…
Adverse events (AEs) are unexpected medical issues that arise upon treatment. Now, this is a very broad term, and it can be broken down into more specific categories depending on…
The American Academy of Neurology (AAN) 2022 Annual Meeting is held from April 2nd until the 7th in Seattle, with its virtual counterpart happening on April 24th to the 26th.…
At the end of March 2022, ALS News Today announced that IFB-088, an experimental treatment for amyotrophic lateral sclerosis (ALS), earned Orphan Drug designation from the FDA. The treatment was…
In 2020, the CDC estimates that there were 241 million cases of malaria globally, of which 627,000 people died. Unfortunately, many of the mortalities were associated with children in sub-Saharan…
Professors Susan Treves and Francesco Zorzato, Basel University researchers, have recently presented the first potential approach to congenital muscle diseases. Their report was published in a recent article by Science…
Currently, no approved therapies exist for ADNP syndrome, a rare genetic neurodevelopmental disorder. But a group of researchers from the UC Davis MIND Institute are working to change that. According…
A retrospective study recently published in the journal Pediatric Rheumatology has detailed the different antiphospholipid syndrome (APS) phenotypes in pediatric patients in effort to develop care approaches which are more personalized. APS APS is…
When two previously independent genes join together, and begin transcribing and translating as if they were one, this is known as a fusion gene. Fusion genes have previously been…
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