Currently, multiple sclerosis (MS), an autoimmune and neurological disorder, is considered either progressive or relapsing-remitting. But could there be additional subtypes? According to new research, yes. As explained in Medical…
As seen in the Journal MDPI, chronic myeloid leukemia only got its name in the mid-1800s, when doctors drew together the category based on the occurrence of a “suppuration of…
The rare liver disease primary sclerosing cholangitis (PSC) may impact just .01% of people, but the extremely pernicious disorder's attacks on the liver mean bearers of the disorder account for…
I was born in 1960 in Chicago, IL and they knew something was wrong seconds after my birth; what, they had no idea. My mother (I was her 3rd child)…
Arimoclomol is a treatment that is in development for ALS, Niemann-Pick disease type C (NPC), and inclusion body myositis (IBM). A recent Phase 2/3 trial has ended in failure, unable…
The Usher 1F Collaborative has recently announced the opening of registration for its scientific conference titled "Therapeutic Strategies for Large Protein Coding Genes in Usher Syndrome." This event will be…
The FDA has recently provided 89bio with guidance for their upcoming clinical trial of BIO89-100, a treatment for nonalcoholic steatohepatitis (NASH). The trial will advance as planned, beginning in the…
At the American Society of Hematology Annual Meeting in 2020 (ASH 2020), the latest info and data related to Gaucher disease, a form of lysosomal storage disease, was presented. It…
According to a story from Cushing's Disease News, the Cushing's Support and Research Foundation (CSRF) is organizing to support Cushing's Awareness Day, which takes place on April 8th. A day…
Steven Elvidge has been left confused, frustrated, and debilitated for the past three years due to a mystery illness. Various symptoms have left him unable to live independently, forcing him…
Each year, researchers make new strides into genetic research, including the discovery of new genetic disorders. According to Medical XPress, researchers from the University of Portsmouth and the University of…
Could something that you're exposed to while still in the womb impact your health? Although it may not be something we think about often, the answer is yes. What we're…
Rare disease patients throughout India have expressed their disappointment and anger towards the country's new policy centered on rare conditions. One of their main concerns is that the policy changes…
The month of April is recognized as Sjögren's Awareness Month, a time for spreading awareness about the autoimmune disease Sjögren's syndrome in the medical community and among the general public.…
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 life, we are faced with a variety of obstacles. But it is the choices we make in facing these obstacles which define ourselves and our legacies. For Avery Allmond,…
At the start of April 2021, the Food and Drug Administration (FDA) approved Regeneron Pharmaceutical's Praluent (alirocumab), a treatment for patients aged 18+ with a rare genetic condition called…
The European Commission (EC) has granted the Orphan designation to Passage Bio for their Krabbe disease treatment, PBKR03. This adds another designation to the medication's list, as it has already…
Since the onset of the COVID-19 pandemic, the novel coronavirus has led to 130 million diagnoses worldwide, with an associated 2.84 million deaths. While doctors and researchers are diligently working…
Connor Dobyn's parents recognized that their son was missing important developmental delays early on. They brought their concern to the doctor, where Connor was diagnosed with autism. For the next…
Each year, researchers make advances in the medical field which assist with the identification, diagnosis, and treatment of patients with rare conditions. According to Genetic Engineering & Biotechnology News, one…
On April 2nd, NFL scouts made their way to Eugene, Oregon to check out some of college football's most talented players. They watched as athletes completed the vertical jump, 40-yard…
According to a recent report in Medical Xpress, over twenty studies have indicated escalating events of nonalcoholic fatty liver disease (NAFLD) in children worldwide. In the United States, NAFLD heads…
Lauren Peikoff’s painful and discouraging experience is unfortunately very common. According to an account reported in NBC’s Think section, Lauren, an associate producer of a national news program, manages…
According to a story from Markets Insider, the biopharmaceutical company CStone Pharmaceuticals has announced that China's National Medical Products Administration (NMPA) has approved avapritinib (marketed as Ayvakit) as a treatment…
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