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…
In the United States, Orphan Drug designation is granted to drugs or biologics intending to treat, prevent, or diagnose rare diseases or conditions. A rare condition is one affecting fewer…
2157 words (source) vs. 423 words (mine) - 5% match In the Phase 2 LILAC clinical trial, researchers sought to evaluate the safety, efficacy, and tolerability of litifilimab (BIIB059) for…
This year, the European Hematology Association (EHA) held its Hybrid Conference starting on June 15; if registered for the conference, you may access the Congress Platform until August 15, 2022.…
A Phase 2 clinical trial which sought to evaluate the safety, efficacy, and tolerability of NP-120 (ifenprodil) for idiopathic pulmonary fibrosis (IPF) had some surprising results. Initially, the study…
Clinical trials are an important tool in medical research to understand more about the safety, efficacy, and tolerability of therapeutics - and how these may influence patient outcomes. Recently,…
Currently, treatment for multiple system atrophy (MSA) is symptomatic; there are no available therapies designed to slow disease progression. This represents a huge unmet need for this patient community.…
Earlier this year, the European Renal Association-European Dialysis and Transplant Association (ERA-EDTA) held its International Congress. During the Congress, researchers discussed the latest in research and scientific innovation within…
Currently, there are few therapeutic options for people who become infected with the Nipah virus. Rather, treatment is symptomatic. However, that may soon be changing. According to the European…
Those living with rare diseases or conditions often face difficulty in finding treatment options - or even spurring researchers to make headway within their disease states. To combat this and…
On July 7, 2022, biotechnology company Akari Therapeutics, Plc ("Akari") shared via news release that the first patient in its clinical trial has completed their course of treatment. Within…
Individuals with Duchenne muscular dystrophy have mutations in the gene that makes the protein dystrophin. This gene is the largest gene in the human body, with 79 exons. When there…
The last approved treatment option for Pompe disease in Europe was given approval in 2006, so 16 years ago. However, the European Commission (EC) recently approved a new therapeutic…
Over the last few decades, there have been immense advancements in medical technology and care options. However, not all of these advancements contribute to better patient outcomes. For example, more…
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