Tags » Rare Disease

Introducing 1st Rare Disease Advocacy World (RDAW) USA 2015 Meeting

Patient advocacy groups are one of the most important powerful stakeholders that contribute to the success of orphan drug development and changes in policies for global regulatory agencies. 354 more words

Orphan Drug Development

Ultra-Rare Diseases: BioBlast Pharma Orphan Drug Designations

BioBlast Pharma, a Tel Aviv, Israel-based clinical-stage biotech founded in 2012, is developing a platform of products for rare and ultra-rare genetic diseases.

I – Cabaletta For Oculopharyngeal Muscular Dystrophy (OPMD) 329 more words

Orphan Drug Development

Umbilical Cords: The Value of Banking and Donating

An umbilical cord is the channel between an unborn baby and its mother — it is also the same thing that saved Marissa Amuso’s life. 633 more words

Blood Disease

How am I doing?

I received a call from my mom the other night asking if I was “ok.” She had read my post about Robin Williams’ suicide and noticed that I seemed low energy a few days before when I took Katherine to meet my parents for breakfast. 470 more words

Rare Disease

Be fair

Once a again a note. All children are excluded from this post as they do not have the full set of coping skills that adults do, as well as the terminally ill and those who suffer from serious mental health disorders. 681 more words


Thank You

There’s always something on my mind.

I mean, like freaking ALLLLLLLLLways.

And that’s why I have this blog.  Where I can write about the crazy… 371 more words

Rare Disease

Top 9 Fastest Growing Orphan Drugs For Past Year

FirstWord Pharma, a supplier of global news and intelligence to the pharmaceutical industry, publishes a list of the top 50 biggest growing drugs for the past 4 quarters (4th Quarter 2013 through the 3rd Quarter 2014). 172 more words

Orphan Drug Development