Jessica Lynn has an educational background in writing and marketing. She firmly believes in the power of writing in amplifying voices, and looks forward to doing so for the rare disease community.
As many patients with rare diseases know, it can sometimes be difficult to achieve the correct diagnosis. In fact, the average length of time it takes from symptom onset to…
In a recent press release, biopharmaceutical company Rallybio announced that the first participant was dosed in a Phase 1/2 clinical trial. Currently, only healthy male participants are enrolled in the…
After announcing positive results from a Phase 2b ADVISE trial, Arena Pharmaceuticals, Inc. ("Arena") hopes to soon initiate a Phase 3 clinical trial to evaluate etrasimod as a treatment…
Recently, biopharmaceutical company Everest Medicines ("Everest") announced positive topline data from the Phase 3 NefIgArd trial, which was run by its licensing partner Calliditas Therapeutics AB ("Calliditas"). Together, the…
According to a new study, researchers believe that lower limb functional surgery improves walking ability and quality of life (QOL) in patients with Charcot-Marie-Tooth disease (CMT). This option provides a…
While clinical trials can be extremely helpful in the development of new treatments, not all trials go as planned. Recently, biotechnology company Regeneron Pharmaceuticals ("Regeneron") discovered this firsthand. As the…
On November 3, 2020, biopharmaceutical company ChemoCentryx and global pharmaceutical company Vifor Pharma Group's sub-business Vifor Fresenius Medical Care Renal Pharma (VFMCRP) announced that the European Medicines Agency will review…
In early November, global biotechnology company BioMarin Pharmaceutical Inc. ("BioMarin") announced the acceptance of its New Drug Application (NDA) for vosoritide. The investigational injectable treatment is designed to treat pediatric…
Later this week, the American Association for the Study of Liver Diseases (AASLD) will hold its virtual The Liver Meeting Digital Experience (TLMdX). Altogether, TLMdX will last from November 13-16.…
In late October, molecular information company Foundation Medicine, Inc. ("Foundation") shared that its diagnostic tool FoundationOne CDx was approved as a companion diagnostic for VITRAKVI (larotrectinib). While FoundationOne CDx will…
For nearly all of 2020, news of COVID-19 dominated the headlines. The first U.S. lockdowns associated with the viral pandemic occurred in March. Now, in November, the novel coronavirus…
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,…
In a recent press release, Catalent and BrainStorm Cell Therapeutics Inc. ("BrainStorm") announced a partnership to manufacture NurOwn for patients with amyotrophic lateral sclerosis (ALS). For over 85 years, Catalent…
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…
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…
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…
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…
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…
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…
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…
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…
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