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…
Often, one's own experiences inspire them to advocate for a certain cause. Abbey Hauser is an example, as she has become a voice for young adults within the rare disease…
Walks are a great way to raise awareness and money for a cause, and they're also good for exercising and spending some time with friends and family. Recently, the Southwest…
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…
Until recently, patients with cyclin-dependent kinase-like 5 (CDKL5) deficiency disorder (CDD) did not have an FDA-approved treatment option. This all changed when the regulatory agency granted approval to ZTALMY, also…
Rare disease research itself is often overlooked in the medical sphere, so it's not difficult to imagine how many singular aspects of rare disease research are forgotten about completely. One…
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…
Cervical dysplasia, a precancerous condition, is defined as "the abnormal growth of cells on the surface of the cervix." While it does not typically cause symptoms, it does lead to…
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…
AstraZeneca has previously submitted a supplemental Biologics License Application (sBLA) for their chronic rhinosinusitis with nasal polyps (CRwNP) treatment, and now, the FDA has responded. In a complete response letter…
The first patient has been dosed in the Phase 1/2 B-LIEVE trial of FLT180a, an investigational treatment for hemophilia B. Freeline Therapeutics, the drug developer, announced the news in a…
Rare diseases can impact many aspects of life, whether that is physical, psychological, educational, or something else. As caregivers are some of the closest people to those with rare diseases,…
It's likely that you've heard of meningitis before - it occurs when the membranes around the brain and spinal cord become inflamed. A number of sources can lead to this…
One thing that we know about psoriatic disease (a term that refers to both psoriatic arthritis and psoriasis) is that is linked to a heightened risk of cardiovascular (CV) mortality…
Because there are so many rare diseases that we still have so much to learn more about, there are many instances where we believe that diseases overlap or are variations…
This Saturday, March 12, marks Narcolepsy Awareness Day, also known as Suddenly Sleepy Saturday. The Narcolepsy Network aims to raise awareness and urges everyone to do their part to advocate…
A recent study conducted at the Penn State College of Medicine focused in on nonalcoholic fatty liver disease (NAFLD), specifically blood clotting in this rare disorder. These researchers knew that…
Karyopharm is currently developing selinexor, an investigational treatment for recurrent or advanced endometrial cancer. They had planned to submit a supplemental New Drug Application (sNDA) to the FDA, but now…
There is a need for more research into Leigh syndrome, as there are currently no FDA-approved treatments, and the patient population faces an unmet need. However, in order for there…
The European Commission (EC) has recently granted its Orphan Drug designation to AR101, a PKCβ inhibitor for the treatment of Ehlers-Danlos syndrome (EDS). This decision was based off of a…
Eosinophilic-associated disorders are a group of conditions characterized by heightened levels of eosinophils, which are a type of white blood cell within the immune system. One of these disorders is…
According to an article from Healio, studies have proven that budesonide oral suspension (BOS) is a viable treatment option for eosinophilic esophagitis (EoE). Research demonstrated that BOS outperformed placebo in…
A new, joint research pact between Illumina and the National Cancer Center Japan aims to shed light onto the clinical information and genomics of nasopharyngeal carcinoma, a rare cancer. Together,…
Policy regarding many topics varies throughout America as one moves from state to state, and rare disease policy is no different. Because of this, getting a holistic view of where…
Cancer is often an avoided topic in conversation - which is understandable. It's scary and uncomfortable to think about, so we like to push it away. And for adolescents and…
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