-
Advance the Research Evolution: Introducing PPMD’s Dystrophinopathy Clinical Research Network
In 1984, the word “Duchenne” entered my world. The progress we have made since then
-
Agamree recommended for NHS use in UK, approved in China
Agamree (vamorolone) has been recommended for use in the National Health Service (NHS) in England,
-
I’ll continue to do what I love, despite the FSHD ‘hangovers’
My wife, Wendy, and I had a wonderful Sunday recently. We rose early and hit
-
$10M in prizes offered toward work on FSHD treatments
The venture philanthropy organization SOLVE FSHD is offering $10 million in prizes for innovators who
-
Catch up on PPMD Together: Virtual Edition – A Gathering of Families for Connection, Support, and Growth
Last month, families from around the world came together for PPMD Together: Virtual Edition. This
-
Arrakis’ RNA-targeted oral therapies showing promise for DM1
Arrakis Therapeutics’ investigational RNA-targeted small molecule (rSM) therapies were found to work as intended in
-
When art and advocacy meet onstage
Last Saturday, I performed a monologue titled “Connection” at the Enabling Lives Festival here in
-
Sarepta to develop Arrowhead’s muscular dystrophy treatments
Sarepta Therapeutics is acquiring from Arrowhead Pharmaceuticals the exclusive global rights to develop ARO-DUX4 and
-
Fashion | Dune London: Bags
With the festive season now in full swing, why not invest in something extra special
-
When the Power of Humanity Expands into the Digital Realm: “The Remarkable Life of Ibelin” Review
“The Remarkable Life of Ibelin” Review by Hawken Miller, Writer and Advocate When Mats Steen,
