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…
According to an article recently published in Targeted Oncology, the FDA approved olaparib (Lynparza), a product of AstraZeneca Pharmaceuticals, in March of this year. Lynparaza will be used as adjuvant…
The EULER 2022 Congress took place this year from June 1st-4th, 2022. During that time, rheumatology professionals met to discuss trends, guidelines, and patient care within the field of rheumatology.…
According to a recent press release from Biospace, the first cohort of the ReSPECT-LM trial has officially completed enrollment. This trial focuses on Rhenium-186 NanoLiposome (186RNL) as a treatment for…
Medscape has recently reported on a new supplemental approval from the FDA for setmelanotide, which is a treatment for obesity associated with Bardet-Biedl syndrome (BBS). Now, BBS patients who are…
In clinical trials evaluating Zolgensma for children with spinal muscular atrophy (SMA), the maximum weight of participants was 18.7 pounds. However, parents with children weighing over that weight might be…
Currently, no approved treatment options exist for children with pediatric low-grade glioma, the most common brain tumor found in children. However, shares Day One Biopharmaceuticals ("Day One") in a news…
According to a story from news-medical.net, the International Rett Syndrome Foundation (IRSF) has announced the launch of its Clinical Trial Committee. The committee was implemented with the goal of helping…
Recently diagnosed acute myeloid leukemia (AML) patients with IDH1 mutations now have a new treatment option, as the FDA has granted approval to TIBSOVO (ivosidenib tablets)! This is an expansion…
Have you ever heard of azithromycin? It’s an antibiotic. Chances are, if you’ve ever had a bacterial infection, you’ve even taken it. But according to Medical XPress, researchers are currently…
In 2022, the 25th Annual Meeting of the American Society of Gene & Cell Therapy took place from May 16th-19th. During the meeting, researchers and other stakeholders discussed the latest…
On May 26, 2022, biopharmaceutical company Nurix Therapeutics, Inc. ("Nurix") shared via news release that positive data was available from an ongoing Phase 1a dose escalation study. Within the study,…
The U.S. Food and Drug Administration (FDA) has recently granted approval to tisagenlecleucel, which is marketed under the name Kymriah, for adult patients with relapsed or refractory (R/R) follicular lymphoma…
The American Academy of Neurology (AAN) held its Annual Meeting from April 22-28, 2022. During the meeting, various stakeholders discussed trends, science, and research within the field of neurology. According…
During the Phase 1/2 SUNRISE clinical trial, which evaluated LB-001 for pediatric patients with methylmalonic acidemia, at least two participants experienced serious and severe adverse reactions: thrombotic microangiopathy. While investigating…
Galecto has announced they have completed enrollment in their Phase2b trial which will investigate GB0139 as a treatment for idiopathic pulmonary fibrosis (IPF). This trial is called GALACTIC-1. Galecto anticipates…
Clinical trials are an important tool within the healthcare realm. These trials assist in developing deeper understandings of diseases, assessing and discovering treatment options, and learning new ways of identification…
A new treatment for Rett syndrome is officially being studied in humans in Canada, meaning Canadian patients may have a new, viable treatment option in the future. Called TSHA-102, this…
Clinical trials are important in developing a better understanding of certain diseases or conditions, as well as how to treat them. According to HCP Live, researchers evaluated PRX-102 (pegunigalsidase alfa),…
According to a recent press release, the Australian Human Research Ethics Committee (HREC) has granted approval to Claritas Pharmaceuticals' submission for R-107. Now, the company is free to move forward with…
According to a recent article in Businesswire, The Lancet has published data from the third phase of the Marigold trial. This clinical trial evaluated ganaxalone - marketed as ZTALMY - as a…
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