According to a story from BioPharma Dive, gene therapy for Fabry disease is still in its early stages of development. However, it is already beginning to show potential as a…
Compassion [kuhm-pash-uhn] noun A feeling of deep sympathy and sorrow for another who is stricken by misfortune, accompanied by a strong desire to alleviate the suffering. Compassion Corner is…
A local radio host and police dispatcher known as Dr. Rock to his audience in Bakersfield, California has recently passed away due to multiple system atrophy (MSA). You may recognize…
The month of August is recognized as Psoriasis Awareness Month, a time of year set aside to help spread awareness about the disease among the general public and in the…
A recent article in Parkinson’s News Today announced that a new study indicates that high blood pressure has been associated with Parkinson’s disease in Singapore’s Han population. This is…
To the vaccinated, it may feel like the COVID-19 pandemic is finally over, but a convergence of the Delta variant, rising long COVID cases, a higher number of breakthrough…
FoundationOne CDx is a comprehensive genomic profiling test that is tissue based. It is used to identify individuals diagnosed with ALK+ non-small-cell lung cancer (NSCLC) who will be eligible for…
A new pooled data analysis of the frontline treatments for metastatic renal cell carcinoma (mRCC) has just been completed. It demonstrated that there is a difference in overall survival for…
Recently, the FDA approved an investigational new drug (IND) application for '1104, a first-in-class peptide for the treatment of eosinophilic esophagitis (EoE). Now, Revolo Biotherapeutics will move forward to investigate…
Sometimes, different surgical techniques are required to operate on adults as opposed to children. For example, surgical revascularization is used to treat pediatric patients with Moyamoya disease - though it…
The month of August is recognized as Stevens-Johnson Syndrome Awareness Month. The goal of this event is to elevate awareness about Stevens-Johnson syndrome among the general public and in the…
The FDA has recently granted Orphan Drug designation to TST1001, Transcenta Holding Limited's monoclonal antibody for gastric cancer/gastroesophageal junction (GC/GEJ). Prior research has demonstrated TST1001's anti-tumor activity, and ongoing research…
In the past, ibrutinib has been used to treat patients with a variety of conditions, including chronic lymphocytic leukemia (CLL), mantle cell lymphoma (MCL), and chronic graft-versus-host disease (cGVHD). According…
Welcome to the Rare Classroom, a new series from Patient Worthy. Rare Classroom is designed for the curious reader who wants to get informed on some of the rarest, most…
In mid-July 2021, biotechnology company Alterity Therapeutics ("Alterity") shared the publication of new data regarding ATH434 for patients with multiple system atrophy (MSA). The data comes from a preclinical…
The month of August has been recognized as Autoinflammatory Awareness Month since 2015. This event is centered on spreading awareness about autoinflammatory illnesses among the medical community and the general…
A recent study documenting cases of pregnancy in individuals living with paroxysmal nocturnal hemoglobinuria (PNH) was published in the Saudi Journal of Medicine & Medical Sciences. Previously, research on pregnancy with…
In a recent news release, rare disease-focused biopharmaceutical company CANbridge Pharmaceuticals Inc. ("CANbridge") shared that the National Medical Products Administration (NMPA) of China accepted an Investigational New Drug (IND) application…
As reported recently in Medscape, forty-eight patients with myotonic dystrophy type 1 (DM1), were identified by way of the DM-Scope registry, which is the largest collection of data for the DM population. The…
Renal cell carcinoma (RCC) is typically treated with single-agent tyrosine kinase inhibitors (TKIs). Now, this may be changing as research demonstrates the potential of the combination of TKIs and immunotherapy.…
“I’ll be honest with you, Lindsey. I had to look up your syndrome before this appointment,” the doctor bashfully admitted. “I have never seen or heard of CLOVES syndrome.” Though…
On August 2, 2021, biotechnology company Amicus Therapeutics ("Amicus") shared that its oral treatment Galafold (migalastat) was approved by the European Commission (EC) for the treatment of adolescent patients (ages…
A new study published in the journal Scientific Reports has helped explain the differences between neuromyelitis optics spectrum disorder (NMOSD) after optic neuritis (ON) and multiple sclerosis (MS). Although previous studies have…
According to a press release from late July 2021, LX1004, a treatment for patients with CLN2 Batten disease, received both Orphan Drug and Rare Pediatric Disease designations from the…
Clinical guidelines are often used to improve patient treatment. These guidelines use evidence reviews, as well as an examination of other standards-of-care, to optimize patient care and outcomes. According…
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