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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 preclinical studies and eased symptoms in a mouse model of myotonic dystrophy type 1 (DM1). The company presented the findings at the Cell Symposia: Chemical Biology in Drugging the Undrugged conference, held earlier this week in San Francisco. “We are…
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When art and advocacy meet onstage
Last Saturday, I performed a monologue titled “Connection” at the Enabling Lives Festival here in Singapore. ART:DIS, a local nonprofit that uses the arts to support people with disabilities, organized the event as part of its BEYOND DIS:PLAY performing arts and theater mentorship program. The festival centered on a development showcase, titled “The Human Condition.”…
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Sarepta to develop Arrowhead’s muscular dystrophy treatments
Sarepta Therapeutics is acquiring from Arrowhead Pharmaceuticals the exclusive global rights to develop ARO-DUX4 and ARO-DM1, two RNA interference (RNAi) therapeutic candidates in Phase 1/2 clinical testing, each for one type of muscular dystrophy. ARO-DUX4 is being tested for facioscapulohumeral muscular dystrophy (FSHD) and ARO-DM1 for myotonic dystrophy type 1 (DM1). Both were designed using…
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Fashion | Dune London: Bags
With the festive season now in full swing, why not invest in something extra special from luxury retailer, Dune London. Whether looking to treat a loved one, or even yourself, I highly recommend checking out their extensive range of quality bags ~ there really is something for everyone and every occasion. (Go on, you know…
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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, a young Norwegian man, passed away from Duchenne muscular dystrophy at 25, his parents Robert and Trude were inconsolable. They had outlived their son, who lived what they saw as an isolated and lonely life due to the physical limitations…
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Couch shopping awakened me to a focus on my children with DMD
I think about things deeply, rolling ideas around in my head until they make sense. My creative side, the writer, likes to wonder in this way. And as an introvert who naturally tends to keep everything to myself, writing is often the way I can express ideas and feelings after they’ve been processed. Reflecting on…
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DMD carrier with heart failure has healthy pregnancy: Case study
A woman who developed heart failure due to Duchenne muscular dystrophy (DMD) was diagnosed and treated before she got pregnant, which led to a healthy outcome for her and her baby girl, according to researchers in Japan. The woman’s case was reported in “Successful Pregnancy Outcome With Preconception Care in a Symptomatic Carrier of Duchenne…
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This Year, I Am Grateful For Our Journey Together
As I prepare for another holiday to begin, I am again overwhelmed with gratitude for the compassionate, resilient community we’ve built through PPMD. You are each an extension of my family, the family we have created together. You bring light and hope for the future as we continue to fight. Over the past three decades,…
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A walker should help prevent falls, right?
I’m getting more comfortable using my walker. However, it just doesn’t pay to trust the darned thing. The other day in my bedroom, I was putting clothes away. I was planning to take a shower later in the day, so I thought I’d save myself some steps. I grabbed a fresh set of clothes and…
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Equitable Access in Clinical Trials—A Call for Action and Collaboration
Living with Duchenne and Becker presents unique challenges, especially for families from underserved or marginalized communities. Health disparities, delayed diagnoses, and limited access to care and clinical research create barriers that often result in diminished clinical outcomes. However, there is a growing sense of hope: by working together across various sectors, we can make meaningful…
