In the past, treatment for progressive familial intrahepatic cholestasis (PFIC) was symptomatic or surgical, meaning that there weren't too many treatment options for this patient population. This all changed with…
Research is constantly being done on possible treatment strategies for different forms of cancer; there are many patient populations that face an unmet need. Currently, a team of researchers from…
It's officially Rare Disease Week, which is being celebrated from February 22nd until March 2nd. Every year, we take this week to to recognize, support, raise awareness, and advocate for…
One of the burdens that rare disease patients often face falls into the financial category, and that's treatments that come at a very high cost. One patient, a young girl…
Mark Hoppus has been working in the music industry for three decades; his band, Blink 182, was formed back in 1992. He has a strong love for writing, performing, and…
Despite undergoing clinical development, many drugs never receive approval from the FDA. Recently, this happened to a treatment for growth hormone deficiency (GHD). The medication, titled somatrogon and owned by…
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As we know, it can be incredibly difficult to receive the proper diagnosis for a rare disease. In fact, it typically takes at least seven years and numerous doctor's appointments.…
A tragedy in Deer Creek, Oklahoma has pushed bacterial meningitis into the spotlight after it claimed the life of Nathan Rogalski. Nathan, who was a star athlete within his community,…
LOXO-305 is an investigational Bruton’s tyrosine kinase (BTK) inhibitor which is currently in development for the treatment of mantle cell lymphoma (MCL). The next step is to evaluate LOXO-305 in…
Misconceptions exist about many rare diseases. In fact, the efforts of many advocates are focused on education and dispelling these myths. Recently, an article in the Hindustan Times did just…
February 1st marked the beginning of Turner Syndrome Awareness Month, which is an entire month dedicated to learning about this rare disease and raising awareness. The Turner Syndrome Foundation (TSF) has…
According to a recent article in SMA News Today, more spinal muscular atrophy (SMA) patients in the United Kingdom (UK) will have access to Zolgensma. This change comes after the…
According to an article recently published in Clinical Trials Arena, the Swedish pharmaceutical company Oncorena has recently initiated a Phase I/II trial for orellanine. This investigational treatment is intended for…
The Memorial Sloan Kettering Cancer Center has recently released skincare guidelines and tips for those living with cutaneous T-cell lymphoma (CTCL). It's important that impacted individuals take good care of…
There are many inequities in our society, and many of these exist within the healthcare sphere. In this article, we're specifically going to look at the inequity that Black women…
Incyte has withdrawn its New Drug Application (NDA) for parsaclisib, which is an investigational treatment for various types of lymphoma, including relapsed/refractory mantle cell lymphoma (MCL), follicular lymphoma, and marginal…
KalVista Pharmaceuticals plans to initiate a Phase 3 trial for KVD900, a treatment for hereditary angioedema (HAE) after finding positive results from earlier Phase 1 and 2 trials. The Phase…
Magnetic resonance imaging (MRI) has been used to study neuromyelitis optica spectrum disorders (NMOSD) in the past. Specifically, it is used to visualize the involvement of the spinal cord's central…
Diagnosing a rare disease early is important, as it allows for prompt intervention and treatment. Recently, researchers took large steps forward in the diagnostics of pulmonary arterial hypertension (PAH) in…
Rare disease patients know what it's like to go without a diagnosis despite enduring symptoms. Doctors often don't recognize the disorders for a number of reasons, whether that's the similarity…
According to a recent article from BusinessWire, the European Commission (EC) has approved KEYTRUDA as a monotherapy for adult patients with renal cell carcinoma (RCC). Specifically, this approval is indicated…
At the end of last year, the FDA warned people with hyperkalemia to monitor their diets when it comes to potassium intake. According to an article from the Taipei Times,…
Our parents can be some of our strongest supporters, and Hawken Miller knows this firsthand. He recently published an article in Muscular Dystrophy News Today detailing the love and support…
Vision loss is a symptom associated with a number of disorders, one of which is Usher syndrome. Medical professionals have spent years trying to fully comprehend the underlying mechanisms of…
Because there are many obstacles that stand in the way of developing drugs for rare diseases, the FDA grants a number of designations to incentivize companies to create these treatments.…
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