Interim results from the Phase 1/2 trial clinical trial investigating CAP-1002 as a treatment for heart disease associated with Duchenne muscular dystrophy (DMD) are being presented today at the 2017 Parent Project Muscular Dystrophy (PPMD) Annual Connect Conference taking place in Chicago. Linda Marbán, president and CEO of Capricor Therapeutics, is talking about the company’s plans…
Parent Project Muscular Dystrophy (PPMD) has added a pre-conference workshop to this year’s 23rd Annual Connect Conference, inviting international medical experts to discuss inflammation and immunity in Duchenne muscular dystrophy (DMD). The conference, set for June 29-July 2, is the largest international conference dedicated entirely to DMD. Each year, the event…
CAP-1002 is a cardiac cell therapy that is currently being investigated in clinical trials for the use of Duchenne muscular dystrophy (DMD)-related cardiomyopathy. MORE: Nine other areas of research for Duchenne MD. The therapy consists of allogenic cardiosphere-derived cells (CDCs), meaning it’s produced in a laboratory using heart tissue…
https://www.youtube.com/watch?v=9z4jkn4Wv3w In this video from MoveForwardPT, a physical therapist (PT) from the Children’s National Medical Center talks about role PTs play in optimizing movement for children with muscular dystrophy (MD) and spinal muscular atrophy (SMA). MORE: The cost of…
Researchers say it is important to carefully assess the timing of the start of corticosteroid treatment for Duchenne muscular dystrophy (DMD) in children and its potential association with disease outcomes. The study, “Associations between timing of corticosteroid treatment initiation and clinical outcomes in Duchenne muscular dystrophy,” was published in the…
Eloxx Pharmaceuticals received a $6 million investment from industry partners to advance a novel therapy for genetic diseases caused by nonsense mutations, including Duchenne muscular dystrophy (DMD), raising the total round of financing to $30 million. The investment will be used to advance the development of ELX-02, the…
Leah Leilani Hello! My name is Leah, and 12 years ago, I was diagnosed with a rare form of muscular dystrophy called mitochondrial myopathy. If you don’t know, mitochondria are the energy machines of the cell. They transform your food into energy so you can live. When…
Results of the Phase 2 MoveDMD trial showed that Catabasis Pharmaceuticals’ candidate drug edasalonexent (CAT-1004) can reduce the rate of functional decline in boys ages 4–7 with Duchenne muscular dystrophy (DMD). The recent findings will…
Speech-language pathologists (SLPs) or speech-language therapists (SLTs) do much more than help people with speech problems such as stuttering or mispronouncing words. For children and adults with neuromuscular disorders like spinal muscular atrophy (SMA), muscular dystrophy (MD) and amyotrophic lateral…
Raxone (idebenone) has been granted temporary scientific approval to be used to treat certain patients with Duchenne muscular dystrophy (DMD) in the United Kingdom (UK). The UK’s Medicines and Healthcare Products Regulatory Agency (MHRA) granted Santhera Pharmaceuticals‘ drug candidate a positive Early Access to Medicines Scheme (EAMS)…
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