Neisseria bacteria colonize on the mucosal surfaces of many animals, including humans. In the past, researchers believed that this bacteria could only cause meningitis and gonorrhea in humans. However, shares…
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 the past, myofibers (terminally differentiated post-mitotic cells that help make up muscle tissue) have been implicated in the development and progression of Duchenne muscular dystrophy (DMD). Basically, researchers have…
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…
It’s no secret that receiving a rare disease diagnosis can take excess amounts of time. Various articles share that, on average, it takes anywhere from four to nine years for…
Right now, there are limited pharmacological therapies available for people with nonalcoholic steatohepatitis (NASH). Rather, treatment options focus on healthy diet and exercise, weight loss, alcohol avoidance, lower cholesterol, and…
Don't forget to read Part 1 of our interview. In Part 1, we discussed what chronic granulomatous disease (CGD) is, how it is treated, and how parents and physicians can identify an…
Don't forget to read Part 1 of our interview, where Todd discusses what acute lymphoblastic leukemia (ALL) is and why it is so important to raise awareness for ALL and other cancers. Today,…
Did you know that September is Childhood Cancer Awareness Month? Although September has just passed, it is still important to raise awareness of the many different forms of childhood cancer…
Over the years, there have been growing concerns about medical research failing to cover or explore diverse groups of people. But in a world where healthcare is not just one-size-fits-all, how…
If you haven't already, don't forget to go and read Part 1 of our interview. In Part 1, David Ogman and I discuss his son Jordan's TECPR2 diagnosis, the diagnostic journey,…
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…
FBXL4-related mitochondrial disease urgently requires new therapies. Unfortunately, this condition is often fatal by early ages (early to mid-childhood). Therefore, it is important to spur research into potential interventions or…
How does CAR T-cell therapy affect an enhanced anti-leukemia effect? Could higher doses influence toxicity or change patient outcomes? According to an article in the Pharmacy Times, researchers sought to…
Glioblastoma, a type of brain cancer, is notoriously difficult to treat. This is because the tumor is fast-moving, aggressive, invasive, and hard to remove with just surgery. However, an…
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…
How is rheumatoid arthritis treated? Currently, the “gold standard” – referring to one of the most effective therapeutic methods – is a combination of methotrexate and adalimumab. This treatment is…
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…
According to an article from Yahoo! Finance, biopharmaceutical company Belite Bio, Inc. has initiated the Phase 3 DRAGON clinical study to evaluate LBS-008 for individuals with Stargardt disease. The…
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…
There are a number of different mosquito-borne illnesses. But sometimes it can be difficult to make an accurate diagnosis when these illnesses – and many others – share similar symptoms…
How do certain conditions contribute to mental health, depression, and anxiety? It's no secret that having a rare, chronic, or underserved condition can be isolating. But what are the actual…
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…
Many patients with ankylosing spondylitis (AS) could be treated with biologics – in particular, biologic disease-modifying antirheumatic drugs (DMARDs). However, this line of treatment is not efficacious nor well-tolerated…
Missense mutations cause Angelman syndrome in around 5% of diagnoses. However, researchers have often been unclear or unsure of the biochemical consequences of these missense mutations on the affected…
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