Drug is first gene therapy for Duchenne muscular dystrophy

A new drug is the first gene therapy approved to treat debilitating and fatal Duchenne muscular dystrophy in four- and five-year-old boys.

Mark Michaud-Rochester • futurity
June 27, 2023 • ~7 min

Daily steroids are safe for kids with Duchenne muscular dystrophy

Daily steroids are not only safe for children with Duchenne muscular dystrophy, they also slow the disease's progression, a new study shows.

Mark Michaud-Rochester • futurity
May 5, 2022 • ~5 min

New gene therapies may soon treat dozens of rare diseases, but million-dollar price tags will put them out of reach for many

New payment models may mean more of the people who need these treatments can get them.

Kevin Doxzen, Hoffmann Postdoctoral Fellow, Arizona State University • conversation
Aug. 31, 2021 • ~8 min

Discovery could lead to Duchenne muscular dystrophy therapies

Research into how chronic inflammation promotes muscle fibrosis could lead to new therapies for people with Duchenne muscular dystrophy, a fatal disease.

Anne Warde-UC Irvine • futurity
April 15, 2021 • ~4 min

New approach prevents muscular dystrophy damage in mice

Giving a boost to molecules involved in muscle repair could help stave off damage due to Duchenne muscular dystrophy, researchers report.

Morgan Sherburne-Michigan • futurity
Feb. 27, 2020 • ~4 min

Protein tweak may prevent DMD-related heart disease

Altering 1 protein in the heart may prevent heart disease related to Duchenne muscular dystrophy, research in mice shows.

Maud Alobawone-Rutgers • futurity
Jan. 15, 2020 • ~2 min

Researchers unveil stem cell models of human spine development

Harvard researchers have unveiled the first stem cell models of human spine development, setting the stage for better understanding of musculoskeletal and metabolic disorders, including congenital scoliosis, muscular dystrophy, and Type 2 diabetes.

Stephanie Dutchen • harvard
Jan. 8, 2020 • ~6 min

CRISPR fix in mice may lead to muscular dystrophy therapy

Using CRISPR to correct mutated muscle stem cells in mice may pave the way for lifelong treatments for people with Duchenne muscular dystrophy.

Derek Thompson-Missouri • futurity
Sept. 19, 2019 • ~4 min

Exercise, fasting shown to help cells shed defective proteins

A new study from the Blavatnik Institute finds that intense exercise and fasting activate hormones that boost cells’ capacity to dispose of defective proteins, which clog up the cell, interfere with its functions, and, over time, precipitate diseases including neurodegenerative conditions such as ALS and Alzheimer’s.

Ekaterina Pesheva • harvard
Feb. 21, 2019 • ~9 min

Can more ‘flags’ help CRISPR treat Duchenne muscular dystrophy?

A new discovery may bring scientists a step closer to using the gene editing technique CRISPR to treat long-term chronic conditions like DMD.

Derek Thompson-Missouri • futurity
Jan. 10, 2019 • ~4 min

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