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…
According to a story from Newswise, a recent study concluded that adult patients living with glomerular diseases face an increased chance of cardiovascular disease as well. The findings were first…
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…
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…
Recently, Docbot, Inc. ("Docbot") announced results from a study performed on Ultivision. Altogether, Docbot's mission has been to create artificial intelligence (AI) technologies that can be applied within a healthcare…
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…
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…
In mid-October, Amolyt Pharma ("Amolyt") announced that the first participant was dosed in a Phase 1 clinical trial evaluating AZP-3601 as a potential treatment for patients with hypoparathyroidism. As of…
Purnell was Taylor and Sam Sabky’s first child, and they couldn’t have been more excited for his arrival and the pregnancy and delivery happened without any major issues. For the…
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…
This year, at the 2020 CNS-ICNA Conjoint Meeting, researchers identified ataluren as a potential treatment that inhibits disease progression for patients with nonsense mutation Duchenne muscular dystrophy (nmDMD). Research suggests…
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…
In late September and early October, the North American Cystic Fibrosis Conference (NACFC) went virtual, as many conferences and symposia this year have been forced to do. However, that does…
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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