The current standard-of-care for Fabry disease is called enzyme replacement therapy (ERT). According to Fabry Disease News, researchers have been working to evaluate an experimental ERT for patients called ISU303.…
According to a late October 2022 news release from gene therapy company AVROBIO, the sixth patient has been dosed in a Phase 1/2 clinical study evaluating AVR-RD-04 for patients…
Glioblastoma multiforme (GBM) is notoriously difficult-to-treat. This cancer is both aggressive and invasive, spreading “tentacles” and becoming hard to remove even with surgery. As a result, finding more effective therapeutic…
According to a news release on Yahoo! Finance, clinical-stage biotechnology company Freeline Therapeutics Holdings plc ("Freeline") shared that it had initiated dosing in the second cohort of the Phase…
According to a late September 2022 news release from biopharmaceutical company AEON Biopharma, Inc., positive topline data is available from a clinical trial evaluating ABP-450 for patients with cervical…
According to an article from Marisa Wexler in Muscular Dystrophy News, promising study data is available from the Phase 1 ARCH study. Within the study, researchers evaluated EDG-5506, an…
In an investor news release from biopharmaceutical company Avidity Biosciences, Inc., the company shared that the FDA placed a partial clinical hold on new enrollment for the Phase 1/2…
In mid-September 2022, specialty pharmaceutical company Acasti Pharma Inc. (“Acasti”) shared via a news release that it had initiated a pharmacokinetic study on GTX-102. Within the study, Acasti hopes…
According to an article in Batten Disease News, enrollment is complete for a Phase 1/2 clinical trial. Within the trial, researchers will be evaluating Batten-1 for adolescents and adults with…
Before you read on, make sure you've checked out Part 1 of our interview, where we discussed what eosinophilic esophagitis is, its symptoms and diagnostic criteria, and why Dr. Dellon chose to…
According to a news release from Revolo Biotherapeutics, enrollment is complete for a Phase 2a proof-of-concept clinical trial. During the study, researchers will evaluate '1104 for eosinophilic esophagitis (EoE), a…
The Phase 3 PLEO-CMT clinical trial evaluated the safety, efficacy, and tolerability of PXT3003 for patients with Charcot-Marie-Tooth disease type 1A (CMT1A). Altogether, 323 people enrolled. During the trial, researchers…
Effective treatment options are crucial for patients with Chagas disease who show serious or life-threatening symptoms. According to Infectious Disease Advisor, a Phase 2 study in its proof-of-concept phase evaluated…
On September 9, 2022, researchers presented data at the European Neuroendocrine Association (ENEA) meeting. According to Yahoo! Finance, one such presentation centered around new safety and efficacy data from the…
As discussed in Part I of this article, a recent HDBUZZ publication featured a news item stating that due to several severe adverse events occurring during the initial AMT-130 trial…
Both Dravet syndrome and Lennox-Gastaut syndrome (LGS) are characterized by frequent and often severe seizures. In many cases, those affected require multiple medications to manage their condition and prevent epileptic…
Have you ever heard of Orphan Drug designation? This designation is granted by the FDA for drugs or biologics intended to treat, diagnose, or prevent rare conditions. Rare conditions…
According to a news release from biopharmaceutical company BridgeBio Pharma, Inc., the first patient was dosed in a Phase 1 study. Researchers are evaluating the safety, tolerability, pharmacodynamics, and pharmacokinetics…
Both the 3rd Annual Gene Therapy for Neurological Disorders meeting and the ADVANCE 2022 Sanfilippo Community Conference were held in July 2022. During both meetings, researchers presented new data from…
For those living with sickle cell disease (SCD), treatment and management can sometimes be difficult. Currently, there is no cure for SCD. Patients may manage their condition using antibiotics,…
Could a vaccine help treat cancer such as Merkel cell carcinoma (MCC)? Immunomic Therapeutics believes so. Its therapeutic plasmid DNA (pDNA) vaccine, ITI-3000, is designed for those with MCC,…
In a news release from biopharmaceutical and RNAi therapeutics company Alnylam Pharmaceuticals, Inc. ("Alnylam"), the company shared that positive data was available from the Phase 3 APOLLO-B clinical trial.…
An article published by Ophthalmology Times Europe shared that the first patient had been dosed in the Phase 2 STARLIGHT clinical trial evaluating MCO-010 as a potential therapeutic option…
Currently, the standards-of-care for treating medulloblastoma include surgical interventions, chemotherapy, and radiation. Finding new and effective treatment options would help to improve patient outcomes. According to an article in The…
According to a news release from biotechnology company Sound Pharmaceuticals, enrollment has begun in the Phase 3 STOPMD-3 clinical trial. So far, the first patient has been enrolled – with…
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