Researchers at the University of California, Davis, have developed an investigational brain-computer interface (BCI) that holds promise for restoring the voices of people who have lost the ability to speak…
HepaRegeniX GmbH has announced the dosing of the first patient in its Phase Ib clinical trial of HRX-215, a novel small-molecule inhibitor designed to promote liver regeneration. This marks a…
Applied Therapeutics has encountered a significant setback in its quest to bring a new treatment to patients with a rare metabolic disorder. According to Biospace.com, the company announced that its…
Myrovlytis Trust, a UK-based charity and Patient Worthy partner, has developed a new resource for those facing osteosarcoma. This devastating cancer often occurs in childhood, teen, and young adult years.…
Novo Nordisk plans to seek approval in the US and the EU this year for their drug Mim8 for use on pediatric patients with Hemophilia A after promising results from…
Spinogenix has reported that its Phase II trial of SPG601 for Fragile X Syndrome (FSX) has met its primary goal, according to Clinical Trials Arena. FXS is a genetic disorder…
As reported by Clinical Trials Arena, Genentech and its subsidiary Roche, provided positive Phase III results with the Food and Drug Administration, or the FDA, of their REGENCY trial (NCT04221477)…
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…
For decades, children battling hereditary angioedema (HAE) faced a challenging reality: preventing life-threatening angioedema attacks meant enduring regular intravenous infusions or subcutaneous injections. This barrier to preventive care has now…
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…
Happy Labor Day Weekend Patient Worthians! We hope you can spend this weekend's extra time with friends and family while gearing up for fall! This week we have a post…
Recently I read an interesting article about a new med that’s been shown to speed the production of blood platelets by as much as 40% which could potentially improve the…
I have two sisters, each with the remarkable talent to walk into a thrift store with only a few bucks and walk out with a brand new wardrobe. Growing up in…
Duchenne muscular dystrophy (DMD) may not sound so bad, but it is. What's so fatal about muscle degeneration? you might ask. Well, the heart is a muscle, so there's that. Luckily,…
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