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What I’ve Learned Since My Son’s LAMA2 MD Diagnosis
Bloom – a Column by Prudence Jones, ColumnsOn Oct. 21, 2019, I sat in a doctor’s office with my 11-month-old son, Alfie, and was handed a piece ... Read more -
MDA Engage Offers Free Educational Events
NewsThrough a program called MDA Engage, the Muscular Dystrophy Association (MDA) is offering virtual educational events for people affected by ... Read more -
#MDA2021 – MDA Virtual Conference to Feature Latest in Clinical Care, Research
MDA 2021, NewsNext week’s 2021 MDA Virtual Clinical and Scientific Conference is expected to attract more than 1,200 clinicians and researchers from across the ... Read more -
I’m Ready to Go Forward After My 2nd Dose of the COVID-19 Vaccine
Columns, Hawk's-Eye View - a Column by Hawken MillerOn Feb. 19, I received my second dose of the Pfizer COVID-19 vaccine. According to the nurse who gave me ... Read more -
Pink, Green, Blue and Purple Lights Illuminated World for Rare Disease Day
News, syndicatedMany diseases have their own awareness color — breast cancer is pink, muscular dystrophy is green, and AIDS is red, ... Read more -
Vamorolone Trial for DMD Finishes Pivotal First Part, Results Awaited
NewsThe final patient has finished the first, six-month portion of the pivotal VISION-DMD Phase 2b clinical trial, assessing the anti-inflammatory ... Read more -
A Hefty Guitar Impacts My Ability to Keep the Music Going
Columns, Of Dragons and Dystrophin – a Column by Andy RuschWhen I was a kid, music from the likes of Led Zeppelin, Fleetwood Mac, and Jim Croce swirled around the ... Read more -
Dosing Begins in Phase 2 Trial Testing Oral Therapy BBP-418 for LGMD2i
NewsThe first patient has been dosed in a Phase 2 trial testing BBP-418, a potential first oral disease-modifying therapy for ... Read more -
Rare Disease Day ‘Patient Hero’ Raises Awareness Through Fashion
News, syndicatedDiagnosed with sickle cell disease as a 6-month-old, Tristan Lee has faced a lot of challenges over his 37 years ... Read more -
Exon Skipping Dominates DMD Treatment, But What’s on Horizon?
NewsWith the recent approval of Amondys 45 (casimersen) by Sarepta Therapeutics, there are now four exon-skipping therapies available in the U.S. ... Read more -
FDA Approves Amondys 45 for Duchenne Patients With Exon 45 Mutation
NewsThe U.S. Food and Drug Administration (FDA) has conditionally approved Amondys 45 — formerly known as casimersen — by Sarepta Therapeutics as the first treatment ... Read more -
Givinostat Continues to Delay DMD Progression in Long-term Study, New Data Show
NewsTreatment with givinostat alongside corticosteroids continues to delay disease progression — and maintain patients’ ability to walk — in ... Read more
