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.
Earlier this week, the FDA approved oral azacitidine (Onureg) for patients with acute myeloid leukemia (AML). According to the ASCO Post, Onureg continuously treats adult patients who previously achieved remission.…
According to Genetic Engineering & Biotechnology News, researchers recently discovered a potential therapy for patients with aggressive subtypes of medulloblastoma. There are four subtypes of this condition. The first is…
Recently, biopharmaceutical company Fortress Biotech announced positive clinical results for CUTX-101 (copper histidinate). The data highlights the efficacy of CUTX-101 for patients with Menkes disease. As there are currently no…
Within the last month, BrainStorm Cell Therapeutics Inc. announced the publication of pre-clinical data on the NurOwn system. NurOwn technology produces autologous, bone marrow-derived mesenchymal stem cells (MSCs) for…
As reported in OncLive, intravenously-administered rigosertib failed to meet the primary endpoint within the Phase 3 INSPIRE clinical trial. Ultimately, the trial sought to understand whether rigosertib could improve overall…
On August 31, 2020, biopharmaceutical company Omeros Corporation announced that it will soon move forward with clinical trials for OMS906. This decision follows clearance of an Investigational New Drug Application…
The 34th Annual Meeting of the Associated Professional Sleep Societies, or "SLEEP 2020," was held last week from August 27th through the 30th. According to BioSpace, pharmaceutical company Harmony Biosciences…
When Jacqueline City went to a concert in high school, she didn't know that her life was about to change forever. At the time, Jacqueline was 18 and living in…
For years, scientists believed there might be a connection between amyotrophic lateral sclerosis and frontotemporal lobar degeneration (FTLD), also sometimes known as frontotemporal dementia. However, researchers from Osaka University may…
Recently, IVX Health announced the opening of three new Tampa-based infusion centers to treat patients with chronic conditions. Currently, IVX Health, who describes themselves as "[providing] biologic injections and infusions…
For patients with rare diseases and their families, life is sometimes isolating. It can be difficult to explain your condition. At the same time, it is incredibly important to find…
Have you ever heard of the Neupro patch? This transdermal delivery system directly delivers a dopamine agonist called rotigotine into the body. For patients with Parkinson's disease, restless legs syndrome,…
There are over 24 million diagnosed cases of COVID-19 worldwide, with over 800,000 associated deaths. The viral pandemic certainly has people feeling upset, confused, and vulnerable. Unfortunately, some resources are…
Earlier this week, Sarepta Therapeutics announced that the FDA approved a New Drug Application (NDA) for Casimersen (SRP-4045). The treatment, for patients with exon 45 amenable Duchenne muscular dystrophy (DMD),…
According to Charcot-Marie-Tooth News, biotechnology company Helixmith recently began a Phase 1/2a clinical trial to test the efficacy of Engensis (VM202) for patients with Charcot-Marie-Tooth disease type 1A (CMT1A). The…
What do microcephaly, Zika virus, Dengue fever, and COVID-19 have in common? According to a recent press release, HP163 could be a treatment for all four. Developed by biopharmaceutical company…
In early June, PatientWorthy wrote about a group of patients called "long haulers." These people, some healthy and some with pre-existing conditions, experienced extended COVID-19 symptoms, sometimes for months after…
Last Tuesday, the FDA rejected BioMarin Pharmaceutical's gene therapy, Roctavian, for patients with hemophilia A. According to ABC News, the gene therapy infusion was meant to be a single treatment…
Last week, biotechnology company Triplet Therapeutics announced their participation in END-DM1, a natural history study to learn more about myotonic dystrophy. There are two major forms of myotonic dystrophy, a rare…
28-year-old Melissa Loewen has always enjoyed helping others. It was this spirit of community that led her to a nursing role in the BC Women’s Hospital's NICU. But a few…
Over the last 2 years, the UK has seen 4 deaths and 320 safety incidents related to missing steroid usage, a story from The Independent explains. For patients with Addison's disease,…
Last week, biopharmaceutical company Deciphera Pharmaceuticals announced results from its Phase 1 clinical trial on QINLOCK (ripretinib). The study, published in the Journal of Clinical Oncology, tested QINLOCK as a potential…
In 2012, a new medical technology emerged: CRISPR-Cas9, which offered the opportunity for gene editing. LabBiotech explains that gene editing could benefit patients with genetic disorders by adding, deleting, or…
Recently, biopharmaceutical company BerGenBio ASA ("BerGenBio") announced that their drug candidate bemcentinib reached its primary efficacy endpoint in a Phase 2 clinical trial. The drug is designed to treat patients…
In a recent press release, biopharmaceutical company Aligos Therapeutics announced that the first patient was dosed in their Phase 1a/b clinical trial. The trial is designed to assess ALG-010133 as…
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