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.
source: pixabay.com
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…
Continue Reading
A New Phase 3 Trial is Being Initiated to Evaluate Etrasimod for Atopic Dermatitis
source: pixabay.com
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…
Continue Reading
Positive Topline Results Reported on Nefecon for IgAN
source: pixabay.com
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…
Continue Reading
Lower Limb Functional Surgery for CMT Improves Walking Ability and Patient QOL
https://pixabay.com/photos/skeletons-funny-hear-no-evil-1617539/
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…
Continue Reading
Garetosmab Dosing Paused in LUMINA-1 Trial for FOP
source: pixabay.com
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…
Continue Reading
EMA Approves MAA Review of Avacopan for ANCA-Associated Vasculitis
Source: pixabay.com
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…
Continue Reading
FDA Accepts Vosoritide NDA for Achondroplasia
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.…
Continue Reading
TLMdX Accepts Late-Breaking Results on Odevixibat for PFIC
Source: Pixabay
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…
Continue Reading
Companion Diagnostics FoundationOne CDx and VITRAKVI Used to Treat Solid Tumors
source: pixabay.com
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…
Continue Reading
Autoantibodies May Cause COVID-19 Blood Clots
https://pixabay.com/photos/student-typing-keyboard-text-woman-849822/
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,…
Continue Reading
ACR Convergence 2020: Presentations on KZR-616 for Autoimmune Conditions
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…
Continue Reading
New Partnership Emerges to Manufacture NurOwn for ALS
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…
Continue Reading
Deep Learning AI Helps Researchers Accurately Diagnose Dystonia
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…
Continue Reading
Could TKIs Be Effective for Patients with Alzheimer’s, Parkinson’s, and Dementia?
source: pixabay.com
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…
Continue Reading
Medical Algorithms Are Harming People of Color
source: pixabay.com
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…
Continue Reading
New Collaboration Will Develop ARO-AAT for A1AD-Related Liver Disease
[Source: pixabay.com]
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…
Continue Reading
Deep Learning Platform Ultivision Can Predict Ulcerative Colitis Severity
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…
Continue Reading
CRISPR CAR-T Therapy is Effective – But is it Safe?
source: pixabay.com
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…
Continue Reading
First Participant Dosed in AZP-3601 Trial for Hypoparathyroidism
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…
Continue Reading
Ataluren Halts Disease Progression in Patients with nmDMD
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…
Continue Reading
Two Poster Presentations on ELX-02 for Cystic Fibrosis Appeared at the NACFC
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…
Continue Reading
AVR-RD-01 for Fabry Disease Granted Orphan Drug Designation
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…
Continue Reading
SX-682 Dosing Complete in Phase 1/2 Trials for MDS, Melanoma
source: pixabay.com
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…
Continue Reading
Scientists Design Synthetic DNA Vaccine for Powassan Virus
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.…
Continue Reading
Ublituximab-Umbralisib Combo for CLL Receives Fast Track Designation
Source: Pixabay.com
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…
Continue Reading
New Study Data Available on Narsoplimab for HSCT-TMA