The collective voice of rare diseases can be heard once again at the upcoming Rare Disease Film Festival
This November, San Fransisco will host the second ‘Disorder: The Rare Disease Film Festival’ screening over 50 films across two days. The...
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Social media is a powerful tool to take rare disease to the masses
When people consider a rare disease they think of cystic fibrosis, as it's taught in schools, or ALS thanks to Stephen Hawkins and the...
Faster new drug approvals, but at what cost?
In 2018, the industry saw a record breaking 56 new drug approvals by the FDA. While 2019 is lagging behind with only 20 approvals, the...
Another step forward in the management of DMD
Duchenne's Muscular Dystrophy is a genetic disorder, mainly affecting boys, and is one of the most common disorders causing progressive...
On the Transformative Potential of Advanced Therapeutics in Rare Disease
The burgeoning field of cell and gene therapies collectively referred to as Advanced Therapeutics (ADVTX), is at last beginning to...
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