Tags » Rare Disease

Sweet Potato Meltdown

I have to cook everything.  Every. thing.

Nothing in our house is processed; it’s too risky. It’s worth it to keep our baby out of the hospital because of accidental ingestion because we missed a label trigger.  403 more words

Europe’s April 2015 Products Recommended For Orphan Drug Designation

The European Medicines Agency (EMA) Committee for Orphan Medicinal Products (COMP) held a meeting April 14 – 16, 2015.

At this meeting, there are 13 positive opinions recommending the following medicines for designation as orphan medicinal products. 269 more words

Orphan Drug Development

Orphan Drug Industry: Top 10 Inspirational Figures In Asia

General lack of understanding of rare diseases, insufficient funding mechanisms and options, absence of legislative frameworks are but a few reasons the Asian orphan drugs market has seen minimal advancements. 150 more words

Orphan Drug Development

Because words

You know how words have power, right?  Words such as, “You didn’t win the contest”, or “You’re going to be a father!” can turn a world upside down.  738 more words

Rare Disease

Findacure: Launch Of Online Portal For Patient Groups

The Orphan Druganaut Blog is honored to have again, Flóra Raffai, the Head of Development from Findacure, as a Guest Blogger. Findacure is a charity which focuses around fundamental diseases – rare, often genetic, diseases from which the basic mechanisms of dysfunction can be extrapolated and applied to our understanding of more common conditions. 381 more words

Rare Diseases

Because Cowden Syndrome means surgeries, apparently

Is there ever going to be a time in my life where surgery isn’t up for discussion?

I really don’t know what to do.  I have had most of the “main” surgeries associated with Cowden Syndrome (thyroidectomy and hysterectomy).  186 more words

Rare Disease

Rare Disease Gene Therapy: Milo Technology’s 1st DMD Patient In Follistatin Trial

Milo Biotechnology is a clinical stage startup company developing treatments to strengthen muscle for neuromuscular diseases. The company is developing a muscle-strengthening Follistatin protein gene therapy for Duchenne Muscular Dystrophy (DMD), in collaboration with… 183 more words

Orphan Drug Development