CureDuchenne will host its annual Futures National Conference, a four-day event focused on bringing education, connection, and hope to the Duchenne and Becker muscular dystrophy (MD) community. The event will be held on May 21-24 at the JW Marriott Orlando, Grande Lakes. It will highlight the latest…
News
The Committee for Medicinal Products for Human Use (CHMP) has recommended that approval of Agamree (vamorolone) in the European Union be expanded to include people with Duchenne muscular dystrophy (DMD) as young as 2. The CHMP is an arm of the European Medicines Agency (EMA) tasked with reviewing…
Starting daily corticosteroids earlier in young children with Duchenne muscular dystrophy (DMD) helps them move better, walk or run faster, and keep their muscles stronger than delaying treatment by about one year, underscoring the importance of early diagnosis for preserving motor function. These are findings from a Phase 3…
Patient-led nonprofit The Speak Foundation has announced the launch of the LGMD Centers of Excellence, a novel alliance of clinics dedicated to improving care for people affected by limb-girdle muscular dystrophy (LGMD). “LGMD has historically faced significant barriers to therapeutic development due to fragmented care infrastructure and extremely…
Upsher-Smith Laboratories has launched two new websites to support people with Duchenne muscular dystrophy (DMD) who are taking its recently introduced corticosteroid therapy Kymbee (deflazacort). One website is designed specifically for caregivers of children with DMD. It offers tips for talking with healthcare providers about treatment, along…
A new collaboration aims to improve the design of clinical trials testing potential treatments for facioscapulohumeral muscular dystrophy (FSHD). The FSHD Society, a patient-driven advocacy organization, is teaming with two other groups — Solve FSHD, a venture philanthropic organization focused on speeding the development of new FSHD treatments,…
The U.S. Food and Drug Administration (FDA) has given Mesoblast the go-ahead to launch a clinical trial testing its cell therapy remestemcel-L-rknd in children with Duchenne muscular dystrophy (DMD). The trial aims to enroll 76 kids with DMD ages 5-9 who are receiving standard-of-care therapies. Participants will…
The Muscular Dystrophy Association (MDA) and Burn Boot Camp, partnering once again for the 10th annual Be Their Muscle campaign, are calling on everyone across the U.S. to join a workout and help raise awareness and funding to support the neuromuscular disease community. Throughout April, more than 400 Burn…
Boys with Duchenne muscular dystrophy (DMD) may be able to regain lost height and overcome growth stunting by switching from traditional steroids to the newer medication Agamree (vamorolone), a new study suggests. While standard corticosteroids are essential for managing DMD-related inflammation, they are known to halt bone…
Bridgebio Pharma has submitted an application asking the U.S. Food and Drug Administration (FDA) to approve its experimental oral therapy, BBP-418, to treat limb-girdle muscular dystrophy type 2i (LGMD2i). If approved, BBP-418 would become the first available treatment for LGMD2i, also called LGMDR9. In fact, according to…
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