Bryan and Bradford Manning recently gave an interview to the Associated Press. The brothers told the story of their vision loss as a result of a rare degenerative eye disease.…
According to a story from PR Newswire, a new collaboration between the Hairy Cell Leukemia Foundation (HCLF) and the Leukemia & Lymphoma Society (LLS) will aim to support critical research…
As reported in CBS News, sisters Izzy and Ailbhe Keane were graced to be born with a natural partner in crime. Their creative flair and eye for style came naturally.…
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…
According to GlobeNewswire, the results from a Phase 1 study of lirentelimab have been released. Allakos developed this medication as a treatment for eosinophilic and mast-cell related diseases. In this…
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…
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…
Charcot-Marie-Tooth (CMT) disease is a neurological disorder with a number of subtypes. Because of this, no treatment is universally successful within all affected individuals. In order to get patients the…
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…
Evrysdi, a treatment for spinal muscular atrophy (SMA), has started to make progress around the world in terms of approval. Brazil's regulatory agency, National Health Surveillance Agency, has approved the…
According to a story from GlobeNewswire, the medication dupilumab (marketed as Dupixent®) has demonstrated potential efficacy in a phase 3 clinical trial as a treatment for eosinophilic esophagitis, a rare…
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…
Medical professionals have been studying inflammatory bowel disease (IBD) for many years in an effort to fully understand it. Now there has been a breakthrough, as researchers from the Children's…
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…
Positive results have just been announced for a Phase 2 study called QUARTZ2, investigating SHR0302 as a therapy for atopic dermatitis (AD). Researchers found that at every dose level examined,…
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…
New Trial A Phase 1A trial for GMA301 was completed this year in Australia for pulmonary arterial hypertension (PAH). This trial demonstrated a positive safety profile for a range of…
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