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…
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…
The mission of biopharmaceutical company TG Therapeutics, Inc. ("TG") has always been to create treatments to fulfill unmet needs for patients. Their focus is on autoimmune disorders and B-cell malignancies.…
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,…
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