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…
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…
Earlier this week, biotechnology company Syntrix Pharmaceuticals ("Syntrix") announced that it had finished dosing all patients in its Phase 1/2 clinical trials. The trials were designed to evaluate SX-682 as a…
A recent study, published in the journal Medicine, has found that Mycopheenolate mofetil (MMF) could be a more advantageous therapy for lupus nephritis than cyclophosphamide (CYC) when it comes to improving serum complement…
While Lyme disease is one of the better known tick-borne illnesses, many people might not know about Powassan virus. This flavivirus, common in both Russia and North America, is found…
Rare Disease Cures Accelerator-Data and Analytics Platform (RDCA-DAP) The RDCA-DAP, funded by the FDA, was first launched in September of 2019. Its aim was to make data more accessible for…
Not all treatments work for every patient, with rheumatoid arthritis (RA) medications being the perfect example. Disease-modifying anti-rheumatic drugs (DMARDs) are the standard of care for RA despite the fact…
Researchers from Nanoscope, thanks to funding by the NIH, have developed a protein that can effectively restore vision in mice. This study was published in Nature Gene Therapy. The Study…
In late October, commercial-stage biopharmaceutical company Omeros Corporation ("Omeros") announced the publication of data from its narsoplimab clinical trial. During the trial, researchers evaluated the therapy as a treatment for…
Recently, IM Therapeutics, a developer of therapies for patients with autoimmune conditions, announced the start of a Phase 1b clinical trial designed to evaluate IMT-002 for type 1 diabetes. Interestingly,…
Many rare diseases are in dire need of need of viable treatments. To fix this problem, researchers have began creating and indicating existing treatments for rare diseases. An example would…
According to a story from PR Newswire, the biopharmaceutical company reVision Therapeutics, Inc. has just announced that its experimental therapy REV-0100 has received both Rare Pediatric Disease designation and Orphan…
An article recently published in Genetic Engineering & Biotechnology News, heralds DrugCell, an experimental artificial intelligence (AI) system. DrugCell was created at the Moores Cancer Center and the School of…
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