According to a story from Charcot-Marie-Tooth News, CMT UK, a United Kingdom-based nonprofit focused on Charcot-Marie-Tooth disease, has released a series of seven exercise videos tailored to patients as part…
According to a story from fiercepharma.com, the US Food and Drug Administration (FDA) was willing to approve the drug Tibsovo as a treatment for acute myeloid leukemia (AML) following encouraging…
According to a story from SELF, Jaime Stathis lives with a rare condition called misophonia, in which certain trigger sounds can cause negative emotional states and outbursts. Jaime discusses how…
In a press release from Sanofi, a global biopharmaceutical company, the company's experimental therapy candidate olipudase alfa has demonstrated the ability to substantially improve spleen volume and lung function in…
Colorectal Cancer (CRC) is now the third leading cause of cancer-related deaths in the U.S. A report in Foundation Medicine highlights data on the increase in the number of cases…
The FDA has recently approved of an updated label for Endari, a treatment for sickle cell disease (SCD). This new label will give medical professionals better information, allowing them to…
A recent study in Frontiers in Neurology published findings on brain changes in patients with Charcot Marie Tooth disease Type 1A, a rare neurological disease that affects the muscles. The researchers have…
Since the company was founded, biotechnology company Kezar Life Sciences ("Kezar") has worked to develop therapies for patients with oncologic and autoimmune disorders. Recently, they highlighted a potential treatment, KZR-616,…
According to a story from Medscape, a recent large scale trial focused on cancer treatment has produced some interesting results. The trial was aimed at testing next generation sequencing of…
Researchers from Rensselaer Polytechnic Institute have been conducting research on tau proteins, specifically how it spreads from cell to cell. According to an article in Newswise, this team of researchers…
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…
According to Neurology Today, researchers have discovered a new way to use artificial intelligence (AI) to enhance medical knowledge. Through the DystoniaNet platform, which uses deep learning AI, researchers targeted…
What sort of treatments are available for patients with dementia and other neurodegenerative conditions? Are these treatments safe and effective? While these questions vary in answers, researchers are currently exploring…
We've seen it time and time again in the news - stories of patients being treated differently, not believed by doctors, or significantly harmed by illnesses simply because of…
In October 2020, Arrowhead Pharmaceuticals ("Arrowhead") announced a new collaboration and licensing agreement with global biopharmaceutical company Takeda Pharmaceutical Company Limited ("Takeda"). Together, the pair will develop and commercialize…
Blood cancer treatment is undergoing a transformation, according to a recent study in Nature Medicine. In this regard, the Leukemia & Lymphoma Society (LLS) recently published its own account featuring…
In late October, CRISPR Therapeutics announced positive top-line data from a Phase 1 clinical trial that was analyzing CTX110 for patients with CD19+ B-cell cancers. However, according to an article…
A recent article published in Multiple Sclerosis News Today reported that newly identified immune cells prevented the death of injured nerve cells. A study finds that this particular immune cell…
A rare disease diagnosis is often shocking, and it means making life changes. An adrenoleukodystrophy (ALD) diagnosis is no exception. Because this condition typically impacts children, parents have to make…
A CNBC News item announced a digital chemistry breakthrough that may revolutionize the entire drug industry. The chemists at Glasgow University’s Cronin Lab have created software that sends out instructions…
As sourced from The Joplin Globe and the Lori Marble Blog, there are a lot of myths surrounding lefties. The minority among us, historically accused of witchcraft and dark magic,…
A series of articles recently published in FierceBiotech describe a discovery by Ohio State and Michigan University scientists. The teams have discovered an immune cell that could be tapped to…
Across the world, various countries have Orphan Drug or Orphan Medicinal Product designations that leading agencies grant to products designed to treat patients with rare diseases. Recently, AVROBIO announced that…
Medical professionals are constantly looking for treatments that will halt or even reverse the progression of Alzheimer's disease, and a new vaccine called E22W42 may be a large step in…
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…
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