Recent research has given us a better understanding of acromegaly, a rare hormonal disorder. Conducted by Maria Fleseriu, MD, FACE, and colleagues at the Pituitary Center at Oregon Health &…
Have you ever heard of PD-L1? The National Cancer Institute explains that PD-L1 is a protein which acts as somewhat of a “brake” for immune response. Sometimes, cancer cells will…
Currently, there are no cures for idiopathic pulmonary fibrosis (IPF), and the two available treatments aim to reduce lung scarring and symptom progression. However, it is clear that new therapeutic…
According to a recent press release from Passage Bio, their investigational gene therapy for Krabbe disease, PBKR03, may be ready to move into the clinical trial stage of drug development.…
Bristol Myers Squibb (BMS) has been developing luspatercept-aamt - marketed as Reblozyl - as a treatment for anemia in non-transfusion dependent (NTD) beta thalassemia. They submitted a supplemental biologics license…
In order to create treatments and diagnostic methods for rare diseases, it's necessary to have a thorough understanding of said disease. A recent study aimed to do just this. Titled…
In the past, there has never been an approved therapeutic option for those with acid sphingomyelinase deficiency (ASMD). This meant that there was a huge unmet need within this patient…
For the first time, a team of researchers measured heart rate variability (HRV) in those with myalgic encephalomyelitis/chronic fatigue syndrome (ME/CFS) to understand how the autonomic nervous system (ANS) responded…
Pancreatic cancer is typically a difficult cancer to treat, leading to poor outcomes and high mortality rates. One of the reasons for this is the difficulty of diagnosis; it often…
Sparsentan is a dual endothelin angiotensin receptor antagonist (DEARA) currently being developed for the treatment of IgA nephropathy (IgAN). The newest update on sparsentan was recently shared by Travere Therapeutics via…
Mitochondria are called the ‘batteries’ that send power to our body’s cells. They convert energy from food into forms that cells can utilize. Mitochondria contain 0.1% of mitochondrial DNA (mtDNA)…
For many rare diseases, the symptoms and experiences of patients are variable. It's extremely rare - if possible at all - to see a rare disease that can be be…
Previous studies have shown that preterm infants (babies born before 37 weeks of pregnancy) often have vitamin D insufficiency. According to Medical Dialogues, researchers in Korea sought to understand the…
Without treatment, an estimated 20% of those with progressive keratoconus may eventually require corneal transplantation. Glaukos, a company creating novel therapies for chronic eye diseases, developed the iLink corneal cross-linking procedure to…
Mutations in the KRAS gene are the most aggressive type of solid tumor. They are responsible for one out of every four cancer deaths. A recent article published by Genentech…
In August of 2021, Intellia Therapeutics of Cambridge and its partner Regeneron Pharmaceuticals published a study reported in the Boston Globe, showing results of a single treatment using CRISPR-Cas9 technology.…
Unintended weight loss is a symptom associated with many different forms of cancer. But what can this weight loss tell researchers about the patients and their outcomes? Healio shares a…
An article recently published in Medical Xpress outlined research into memory recall in Rett syndrome patients. Not only did it look into the underlying causes of diminished memory recall, but…
According to a recent article, although the number of ticks are rising in Germany currently, the cases of tick-borne encephalitis have been surprisingly lower. Researchers found that this is due…
When it comes to finding treatments for diseases, whether they're common or rare, the process doesn't always start from scratch. In many cases, existing drugs are taken and studied in…
Currently, 95% of rare diseases don't have an effective treatment. Progress is being made, but there is just so much ground to cover in rare disease research. Studying rare diseases…
A research study led by Professor Beishi Zheng from Columbia University has studied the immunogenetics of gastrointestinal (GI) cancers. The Study This study demonstrated that individuals who are diagnosed with…
Diagnosing certain conditions can be difficult for many reasons, one of which is the similarity between symptoms of numerous diseases. Doctors can mistake one disease for another, which then impacts…
Did you know that, currently, there are vaccinations to confer protection against cholera? However, there are difficulties associated with this vaccine. For example, current vaccines are relatively ineffective in young…
Have you ever heard of Orphan Drug designation before? The FDA grants Orphan Drug designation to drugs or biologics intended to treat, diagnose, or prevent rare diseases or conditions. In America,…
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