Do you know about Lafora Disease? This rare genetic disease begins during adolescence. Your child starts their life healthy and whole, but then they decline, cognitively and physically, until they…
The results released from the Phase III ESSENCE clinical trial, and recently published by Medical Xpress and in the NEJM, found that the drug semaglutide is effective in the treatment…
Humans have been using tobacco as a drug for more than 12,000 years. It wasn't until the early-20th century that the link between tobacco and lung cancer (and other health problems)…
Approximately one and one half million people are affected by ulcerative colitis (UC) worldwide. It is an inflammatory bowel disorder with, as of yet, no known cause. There are, however,…
Imagine that your loved one is diagnosed with a rare disease. The first thing you do is to go to the internet to understand what you can about this disease…
By: Harsha K Rajasimha, Ph.D., Founder and CEO of Jeeva Clinical Trials Challenges Unique to Cell and Gene Therapies (CGTx) Clinical Trials Cell and gene therapies (CGTx) have emerged as…
Rare disease drug development can be challenging. Countries globally developed programs to incentivize innovation and development in this space. One of these initiatives is the designation of orphan drugs, or…
Clinical-stage biopharmaceutical company Savara Inc. was founded on a desire to develop innovative therapies for people living with rare respiratory diseases. The company's work over the past few years centers…
Ocugen, Inc., a biotechnology company based in Malvern, Pa. recently reported to BioSpace that DSMB gave a favorable review of its Phase ½ ArMaDa clinical study of OCU410. Approximately 10…
Long COVID is a phenomenon emerging from the global spread of SARS-CoV-2, a strain of coronavirus causing COVID-19. Seven million people died from COVID-19, though this is considered a serious…
Mirum Pharmaceuticals has long been a leader in the rare liver disease space, driving forward with a mission of empowering patients and families, and identifying novel therapeutic interventions to help…
Three years after the U.S. Food and Drug Administration (FDA) first placed a full clinical hold on Larimar Therapeutics’ Phase 2 clinical study, the hold has officially been lifted. The…
Artificial intelligence (AI) is changing the way we live. It seems rare to find an industry where AI has not made at least a preliminary impact. In politics, AI-generated cartoons…
An estimated 25% of people with Clostridioides difficile (C. diff) infections experience recurrence following successful antibiotic intervention. After three or more recurrent infections, the risk of developing another infection sits…
People with idiopathic pulmonary fibrosis (IPF) already have two FDA-approved treatment options: Ofev (nintedanib) and Esbriet (pirfenidone). But biotech company Agomab Therapeutics ("Agomab") is working to introduce another potential therapeutic…
You might have heard of Orphan Drug designation - and maybe even Rare Pediatric Disease designation. But have you heard of Regenerative Medicine Advanced Therapy (RMAT) designation? Described in Section…
The Phase 1b/2a RESOLVE clinical study set out to identify how safe and effective EP-104GI is for people living with a chronic immune-mediated disease called eosinophilic esophagitis (EoE). People with…
Just about one month ago, clinical-stage biotechnology company AnaptysBio announced the availability of positive top-line results from two Phase 3 clinical studies: GEMINI-1 and GEMINI-2. Both studies sought to assess…
RINVOQ (upadacitinib) is a Janus Kinase inhibitor that is currently approved to treat a variety of conditions: moderate to severe atopic dermatitis, active psoriatis arthritis, moderate to severe rheumatoid arthritis,…
The American Society of Gene and Cell Therapy (ASGCT) held its 27th Annual Meeting from May 7-11, 2024. During the conference, stakeholders in the field of cell therapy come together…
When it comes to medical research, diversity, equity, and inclusion need to be larger parts of the conversation. As it stands now, medical research can be fiercely hierarchical, expensive, and homogenous in terms of…
Do you know what your macula does? The macula, or the central portion of your retina, plays a role in your central vision; this is required for reading, facial recognition,…
Currently, there are no approved treatment options for myotonic dystrophy type 1 (DM1), a progressive neuromuscular disease. As the disease progresses, affected individuals experience muscle weakness, respiratory distress, and cardiac…
Type 1 narcolepsy, formerly known as narcolepsy with cataplexy, is a chronic neurological disorder characterized by excessive daytime sleepiness and cataplexy, or the sudden loss of muscle tone that is…
Biopharmaceutical company Inozyme Pharma ("Inozyme") has become a leader at the forefront of developing innovative therapies for rare diseases such as ENPP1 deficiency (the childhood form is known as generalized…
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