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July 15, 2019July 15, 2019

Exondys 51 Superior to Glucocorticoids in Preserving Respiratory Health of Duchenne Boys, Analysis Shows

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Treatment with Exondys 51 (eteplirsen) significantly slows respiratory decline in boys with Duchenne muscular dystrophy (DMD), including those with more advanced disease, compared to standard glucocorticoid therapy, an analysis of clinical trials ... Read more

July 12, 2019July 12, 2019

$500K Gift to MDA Summer Camp Will Honor Robert Bennett Legacy

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For more than 20 years, Robert (Bob) Bennett was a steadfast leader and supporter of the Muscular Dystrophy Association (MDA). To honor his legacy, the Robert M. & Marjie ... Read more

July 10, 2019

Macedonian Gaucher Activist Publicizes Plight of Rare Disease Patients

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The challenges Vesna Aleksovska faced when she decided a decade ago to help fellow Macedonians with rare diseases were so daunting, they would have scared off all but the ... Read more

July 10, 2019July 10, 2019

Potential DMD Treatment, Pamrevlumab, Helps to Preserve Lung, Heart and Upper Limb Function in Youth, Early Trial Data Show

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Early trial data supports the potential of FibroGen‘s investigative treatment pamrevlumab to slow the decline in, or even improve, lung and heart function in non-ambulatory youth with Duchenne muscular dystrophy ... Read more

July 8, 2019July 8, 2019

MDA Awards Locana $550K to Advance Potential RNA-targeting Therapy for Myotonic Dystrophy

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The Muscular Dystrophy Association (MDA) has awarded Locana $550,000 through its MDA Venture Philanthropy (MVP) program to help advance the biotech company’s therapy platform for myotonic dystrophy (DM). Specifically, the grant ... Read more

July 4, 2019

World’s First Alport Stamp Is Macedonian Mom’s Latest Win for Rare Disease Patients

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It wasn’t until Gordana Loleska’s son David was 14 years old that doctors in their native North Macedonia diagnosed his kidney, vision, and hearing problems as Alport syndrome. Although ... Read more

July 3, 2019July 3, 2019

Raising Levels of Protein, Sarcospan, Seen to Benefit Cardiac Muscles in Mouse Model of DMD

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Increasing the amount of a protein called sarcospan may help to protect against heart failure in children with Duchenne muscular dystrophy (DMD), a study in mice suggests. The study, ... Read more

July 1, 2019July 1, 2019

Enrollment Progressing Quickly in PolarisDMD Trial Testing Edasalonexent, Catabasis Says

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Enrollment is progressing quickly in Catabasis Pharmaceuticals‘ pivotal Phase 3 PolarisDMD trial, testing its investigational small molecule therapy edasalonexent for Duchenne muscular dystrophy (DMD) — with sites in some countries ... Read more

June 28, 2019June 28, 2019

Edasalonexent, Potential DMD Therapy in Phase 3 Trial, Seen to Preserve Bone Health in Test in Mice Against Corticosteroid

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Catabasis Pharmaceuticals announced new preclinical data showing that, in a mouse model of Duchenne muscular dystrophy (DMD), the small molecule edasalonexent preserved bone health, whereas the corticosteroid prednisolone caused bone ... Read more

June 26, 2019June 26, 2019

NORD Honors Industry, Patient Advocates at Rare Impact Awards Gala

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A violinist with vasculitis, two Texas politicians and a pharmaceutical company whose marijuana-derived therapy helps kids with Dravet syndrome were among winners of the 2019 Rare Impact Awards. Officials ... Read more

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