Category Archives: Muscular Dystrophy Treatment

Market Dynamics of Duchenne Muscular Dystrophy (DMD) Therapeutics Market: Market Driver: high unmet need Market Trend: increasing research funding Market Challenge: high treatment costs. High unmet need. Continue reading →

(Credit: Unsplash) This article is brought to you thanks to the collaboration ofThe European Stingwith theWorld Economic Forum. Author: Sean Fleming, Senior Writer, Formative Content More than 300 million people worldwide live with rare diseases, for which there are no preventative measures, no cures, and no effective treatments. There are about 7,000 different rare diseases. Continue reading →

In 2018, Pfizer initiated a Phase Ib clinical trial of its gene therapy treatment for Duchenne muscular dystrophy (DMD). The AAV9 gene therapy, PF-06939926, is designed to provide DMD patients with a form of the dystrophin gene at the root of the condition, thereby countering the progressive muscle degeneration and weakness that characterizes the disease. Pfizer planned to dose 15 patients with the gene therapy, which must be stored at -70C. Continue reading →

Personalized medicine has taken a big step forward with the launch of non-profit n-Lorem Foundation, which will create patient-tailored antisense oligonucleotide (ASO) therapeutics for people with rare diseases at no cost to the patients. This comes at the same time as custom gene therapies for rare disease patients are being developed, including some combined with CRISPR. Continue reading →

As we mark the start of the 2020s, its interesting to reflect on the advances weve seen in research into muscle-wasting conditions over the past decade and what the next 10 years might bring. Continue reading →

-Enrollment ongoing in clinical trials of CTX001 for patients with severe hemoglobinopathies- -Enrollment ongoing in clinical trial of CTX110, targeting CD19+ malignancies- -Enrollment has begun in clinical trial of CTX120, targeting B-cell maturation antigen (BCMA)- ZUG, Switzerland and CAMBRIDGE, Mass., Feb. 12, 2020 (GLOBE NEWSWIRE) -- CRISPR Therapeutics(CRSP), a biopharmaceutical company focused on creating transformative gene-based medicines for serious diseases, today reported financial results for the fourth quarter and full year ended December 31, 2019. In 2019, CRISPR Therapeutics achieved important milestones and momentum across key programs. Continue reading →

ANN ARBORA team led by University of Michigan researchers has found that using drug compounds to target specific molecules within muscle cells can ameliorate Duchenne muscular dystrophy in mice. Those who have Duchenne muscular dystrophy have a genetic flaw that causes muscle membrane damage, preventing muscle cells from repairing themselves, according to recent research. Now, U-M researchers have found that targeting a string of molecules called a calcium ion channel in the lysosomes of muscle cells can increase membrane repair, ameliorating muscular dystrophy in mice afflicted with the disease Continue reading →

BOSTON--(BUSINESS WIRE)--Catabasis Pharmaceuticals, Inc. (Catabasis, the Company, we, our, or us) (NASDAQ: CATB), a clinical-stage biopharmaceutical company, today announced the closing of its previously announced underwritten public offering of an aggregate of 5,290,000 shares of common stock at a public offering price of $5.00 per share, including 690,000 shares issued upon the exercise in full by the underwriter of its over-allotment option. Continue reading →

Testing a new therapeutic in human subjects for the first time is a major step in the translation of any novel treatment from the laboratory bench to clinical use. When the therapeutic represents a paradigm shift, reaching this milestone is even more significant. After years of planning, preparation and hard work to establish a base camp, starting human clinical trials is the first step towards the summit itself: gaining regulatory approval for product sales. Continue reading →

SOUTH PLAINFIELD, N.J., Feb. 3, 2020 /PRNewswire/ --PTC Therapeutics, Inc. (NASDAQ:PTCT) today announced real-world data showing that boys with nonsense mutation Duchenne muscular dystrophy treated with Translarna (ataluren) and standard of care (SoC), preserved the ability to walk for years longer than those on SoC alone. Continue reading →