Recently, research and development (R&D) company Neurophth Therapeutics announced that the FDA granted Orphan Drug designation to its therapy NR082. Also known as rAAV2-ND4 or NFS-01 project, NR082 is…
According to a recent press release, Japan just approved ULTOMIRIS (ravulizumab) for patients with atypical hemolytic uremic syndrome (aHUS). Developed by biopharmaceutical company Alexion Pharmaceuticals ("Alexion"), ULTOMIRIS represents a…
by Lauren Taylor from In The Cloud Copy Spinal muscular atrophy or SMA is a genetic disease that affects the central and peripheral nervous systems, as well as voluntary muscle…
For many years, researchers linked obesity to a number of other health conditions. Now, Taiwanese researchers identify a new cause for concern: worsening health outcomes for patients with ankylosing…
Glut1 Virtual Community Gatherings: Australia & New Zealand September 27th, 2020 Patient Voice in Research - input discussions for survey development The Glut1 Deficiency Foundation is not letting the coronavirus/COVID-19 pandemic…
Glut1 Virtual Community Gatherings: Children with Glut1 Deficiency Glut1 Pals September 26th, 2020 The Glut1 Deficiency Foundation is not letting the coronavirus/COVID-19 pandemic stop them from continuing in their mission…
Glut1 Virtual Community Gatherings: Glut1 Deficiency Parents Patient Voice in Research - input for survey development to capture patient and family experiences and identify needs September 26th, 2020 The Glut1…
Disclaimer: This blog post has been republished with permission from the author. To visit the original blog site, click here. Written by Tamara Fowler Where do I begin? Thank you…
CRISPR Therapeutics, in combination with Vertex Pharmaceuticals, has recently announced that the European Medicines Agency (EMA) has granted the Priority Medicines (PRIME) designation for their sickle cell disease (SCD) treatment,…
According to a story from Charcot-Marie-Tooth News, an experimental HDAC6 inhibitor called CKD-504 appeared to restore myelination in mouse and cell models of Charcot-Marie-Tooth disease type 1A in a recent…
Disclaimer: This blog post has been republished with permission from the author. To visit the original blog site, click here. Written by Tamara Fowler The weekend of June 11, 2011,…
Turning 18 means many things: the right to vote, buy a lottery ticket, join the military, and even go skydiving. That transition into independence gives freedom, but also newfound responsibility.…
According to a story from Medpage Today, the Society of Hematologic Oncology recently held its virtual meeting on September 9-12. During the event, Dr. Srdan Verstovsek of the MD Anderson…
Dr. Richard Furie at New York’s Feinstein Institutes recently interviewed with MedPage Today. Dr. Furie discussed the prognosis for lupus nephritis and belimumab (Benlysta), a recently-tested drug that appears…
Did you know that testing protein levels in our bodies can tell researchers about our health? In some cases, it can even give insight into disease progression and development.…
According to a story from BioSpace, the biopharmaceutical company Bristol Myers Squibb Canada recently announced that its medication fedratinib (marketed as INREBIC®) has been approved by Health Canada as a…
A new treatment for acromegaly has recently become available for patients. Called MYCAPSSA, this delayed-release capsule is intended for long term maintenance of acromegaly. Considering that injections are the typical…
Graves' Disease Awareness Month is recognized in July of each year. As part of this year's awareness campaign, the nonprofit organization Prevent Blindness and Horizon Therapeutics collaborated to create the…
According to an article from BioTech 365, data from the Phase 3 INVICTUS trial and Phase 1 dose-escalation study, which evaluated QINLOCK as a treatment for gastrointestinal stromal tumors, was…
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…
According to a story from PR Newswire, the RNA-targeted gene therapy company Locanabio, Inc. has recently released findings from a preclinical evaluation of an RNA-targeted CRISPR Cas9 (RCas9) system as…
This past week, Cycle Pharma announced FDA approval of a once daily dose of NITYR for patients with hereditary tyrosinemia type 1 (HT-1). According to its prescribing information, NITYR…
Cynata Therapeutics Limited has recently announced the results from their preclinical trial of induced pluripotent stem cell (iPSC)-derived Cymerus™ mesenchymal stem cells (MSCs), according to BioSpace. They are testing these…
A recent study published in the journal Blood Advances has documented that hematopoietic stem cell transplantation (HSCT) is both safe and effective for children with many different kinds of inherited nonmalignant conditions…
The American College of Obstetricians and Gynecologists (ACOG) has established new recommendations for pregnant women, including perinatal screening for depression as well as anxiety and mood disorders. A total of 40…
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