Pharnext has released an update on the clinical development of PXT3003, a treatment for Charcot-Marie-Tooth disease type 1A (CMT1A). According to the biopharmaceutical company, they are on track to dose…
As reported in News Wise, these oncologists don’t like their options. They want to provide patients with the best possible options to tackle the lethal cancer mesothelioma, but their hands…
According to a story from Market Watch, the gene therapy company AVROBIO, Inc. has recently announced that its investigational gene therapy candidate AVR-RD-04 has earned Orphan Drug designation from the…
In 2015, the FDA approved Apollo Endosurgery, Inc.'s Orbera Intragastric Balloon for weight loss. Now, the company shares in a press release, the device recently received Breakthrough Device Designation…
Some cancers and hematological conditions can be difficult to treat. But biotechnology company Oncopeptides AB is working to address this issue for patients with relapsed or refractory (R/R) multiple…
According to BioSpace, Kronos Bio will continue with its plans for a Phase 3 trial of entospletinib after a positive meeting with the FDA. One of the meeting's focuses was…
For patients with severe atopic dermatitis, it can be difficult to find adequate treatment options. For many, treatment revolves around topical creams, anti-inflammatory medications, light therapy, antibiotics, and more. But…
Researchers from Northwestern University have discovered a compound with the ability to stop the progressive degeneration of motor neurons in those with amyotrophic lateral sclerosis (ALS). The continued breakdown of…
During the National Institutes of Health (NIH) Rare Disease Day at NIH event, a panel was held titled "From Kitchen Tables to Changing Paradigms — Advocacy as a Driving Force…
In a joint statement, biopharmaceutical companies AstraZeneca and Merck & Co., Inc. ("Merck") shared that LYNPARZA (olaparib) achieved a superiority boundary in the Phase 3 OlympiA clinical trial. During…
The National Institutes of Health (NIH) held its annual Rare Disease Day event on March 1, 2021 this year. This event showcases groundbreaking research, amazing rare patient stories, and more.…
Pharnext has announced that they will soon open enrollment for their PREMIER trial of PXT3003, a Charcot-Marie-Tooth disease type 1A (CMT1A) treatment. Beginning in March, researchers will search for 350…
According to GlobeNewswire, Tiziana Life Sciences and Parexel Biotech have agreed to collaborate on a Phase 1b/2 trial of foralumab, an oral treatment for Crohn's disease. As previous research has…
The Brazilian Health Regulatory Agency, ANVISA, has granted market approval to Insightec for their focused ultrasound, Exablate 4000. This MR-guided ultrasound is used for the treatment of tremors that define…
Billions of dollars have been spent in research over decades, resulting in only two therapies that treat symptoms of Alzheimer’s disease (AD). There are no approved methods that diminish the…
In some cases, elderly patients (those over 70 years old) with esophageal cancer have been told that surgery is not the best option. This stems from a belief that…
According to a story from One News Page, the biopharmaceutical company BridgeBio Pharma and its affiliate ML Bio Solutions have recently announced that dosing has begun in its phase 2…
According to a story from businesswire.com, the biopharmaceutical company Ipsen recently presented a total of 9 study abstracts at the recent 18th annual conference of the European Neuroendocrine Tumor Society…
From February 25-27, the Americas Committee for Treatment and Research in Multiple Sclerosis will run its ACRTIMS Forum 2021, according to BioSpace. At this forum, Clene Inc. will present interim…
A Phase 3 study of upadacitinib, an ulcerative colitis treatment, met both its primary and secondary endpoints, according to an announcement from AbbVie. The trial, titled U-ACCOMPLISH, saw a third…
Raghav is a two-year-old boy born with an ultra-rare disease caused by a mutated GPX4 gene. He and eight other children are the only ones in the world who have…
According to a story from The Whitewater Company, parents Paul & Heather Lynn were at a loss for what to do when their daughter Clarity was born with the rare…
Policymakers Must Offer Solutions That Provide Patients with Lower Out-of-Pocket Costs For Critical Medicine In addition to coping with chronic illness, many patients are faced with the challenge of…
In early February, global biopharmaceutical company Sanofi and biotechnology company Regeneron Pharmaceuticals, Inc. ("Regeneron") shared that its dually developed drug Libtayo (cemiplimab-rwlc) recently received FDA approval. Now, Libtayo stands as…
Biotech company Ovid has released disappointing results from a trial of OV101, a potential treatment for Angelman's syndrome. The Phase 3 trial demonstrated that there was no significant difference in…
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