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…
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…
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