Amolyt Pharma has recently announced that they have dosed the first patient in their trial of AZP-3601, a treatment for hypoparathyroidism. According to GlobeNewswire, this treatment will adequately address the…
According to a story from GlobeNewswire, the medical device company SoniVie announced recently that the US Food and Drug Administration (FDA) has granted approval for its Investigational Device Exemption (IDE).…
According to a story from Market Watch, the biopharmaceutical company Alexion Pharmaceuticals, Inc. has recently announced that the US Food and Drug Administration (FDA) has approved a new formulation of…
In early October, global biopharmaceutical company Bristol Myers Squibb announced the Canadian approval of ZEPOSIA (ozanimod) for patients with multiple sclerosis (MS). In particular, the drug is designed to…
A team of scientists have created a nanoparticle tool which allows scientists across the country to study COVID-19 without any risk of infection. This particle mimics how the virus infects…
According to a story from GlobeNewswire, the biopharmaceutical company Immunomedics, Inc., has recently announced that its medication sacituzumab govitecan-hziy (marketed as Trodelvy) has recently earned Orphan Drug designation from the…
Ionis Pharmaceuticals has recently released the data from their clinical trial of IONIS-ENAC-2.5Rx, a treatment for cystic fibrosis. According to Newswire, the study showed very positive results, with healthy volunteers…
According to a story from GlobeNewswire, the gene therapy company Axovant Gene Therapies Ltd. recently announced that its gene therapy AXO-AAV-GM2 has been given Rare Pediatric Disease designation by the…
Last week, clinical stage biopharmaceutical company Neurotrope announced that the first patient was dosed in a Phase 2 clinical trial to test the safety, efficacy, and tolerability of bryostatin-1…
Grifols, a pharmaceutical company based out of Barcelona, Spain, has announced that their potential treatment for the novel coronavirus has entered a phase 3 trial, according to BioSpace. It is…
According to a recent article in BioSpace, young children and infants who have been diagnosed with primary hyperoxaluria type 1 (PH1) are often faced with the use of gastrostomy…
According to a story from GlobeNewswire, the biopharmaceutical company X4 Pharmaceuticals, Inc. has just announced that the US Food and Drug Administration (FDA) has granted its investigational product candidate mavorixafor…
The receptors in the human epidermal growth factor receptor 2 (the HER2 ) gene are active participants in determining whether healthy breasts can repair themselves. According to a recent article…
According to a story from Pharma Advancement, Bristol Myers Squibb recently announced encouraging findings from a phase 3 clinical trial. This study was testing its drug ozanimod (marketed as Zeposia)…
Waking up to the news has one encouraging note, another new day and another new drug is being tested. According to an article in Biospace, the news from Sorrento Therapeutics…
The FDA has recently granted the Fast Track designation to AGLE-102, a treatment for dystrophic epidermolysis bullosa. This designation will allow for the treatment to reach patients at a quicker…
According to a story from Muscular Dystrophy News Today, a gene therapy in development by the pharma company Pfizer, Inc. was recently given Fast Track designation from the US Food…
The FDA has recently granted the Breakthrough Therapy designation to FARXIGA, a chronic kidney disease treatment created by AstraZeneca. This designation will allow for a quicker development, which will hopefully…
Fulcrum Therapeutics is beginning a Phase 1 trial of FTX-6058, a treatment for sickle cell disease (SCD). Researchers will focus on the tolerability, safety, and pharmacokinetics of the medication. The…
According to a story from PR Newswire, the biotechnology company REGENXBIO Inc. has recently announced plans to expand its RGX-121 developmental program. RGX-121 is being developed as a potential one-time…
According to a recent report in the Journal of Clinical Oncology, over one thousand patients with high-risk stage III melanoma were included in the Keynote-054 clinical trial (NCT02362594). The…
According to a story from Targeted Oncology, an experimental combination therapy tested on patients with advanced clear cell renal cell carcinoma (ccRCC) appeared to show potential for efficacy in a…
In late September, GlaxoSmithKline announced FDA approval of its treatment, Nucala, for patients with hypereosinophilic syndrome (HES). The treatment is safe for patients older than 12. Additionally, this approval is…
As written in BioSpace, Genetech has recently presented new two-year data on their spinal muscular atrophy (SMA) treatment, evrysdi. The information was released at the 25th International Annual Congress of…
The FDA has recently granted two designations, the rare pediatric disease and orphan drug designations, to Spirovant Sciences' gene therapy for cystic fibrosis (CF). SPIRO-2101 will now be able to…
© Copyright 2024 Patient Worthy
Sign Up With a Patient Worthy Account and Share Your Rare Story
- OR -
Make a difference, share your experiences and get paid. Opt-in and join Patient Worthy's panel for paid opportunities such as surveys, market research, patient advisory panels and more.
- OR -
Make a difference, share your experiences and get paid. Opt-in and join Patient Worthy's panel for paid opportunities such as surveys, market research, patient advisory panels and more.
