Ensuring that the worlds of teens and adults with Duchenne 'grow bigger', as they grow older by expanding their reach – both literally and figura…
PPMD has awarded a $600,000 grant to the New Jersey Institute of Technology (NJIT) and Talem Technologies as part of our ongoing exploration of robotic technology to assist people living with Duchenne.
“Genetic testing is essential in diagnosing rare diseases, but the combination of genetic information with patient-reported data has potential well beyond diagnosis to help advance the understanding and treatment of these diseases,” said Pat Furlong, founding president and CEO of Parent Project Muscular Dystrophy. “We are heartened to see AltaVoice and Invitae joining forces to help forge new ways to connect patients to care and to contribute to vital research.”
Frequently, I get calls from mothers/sisters/aunts of people living with Duchenne about issues related to being a carrier of Duchenne:
Should they have carr…
There is widespread consensus that female carriers of Duchenne are at risk for symptoms of muscular dystrophy. But what are those risks? PPMD is excited to support this important study at Nationwide Children’s Hospital, which will evaluate the effects of a lower level of dystrophin in female carriers in three meaningful areas—cardiac, musculoskeletal, and psychosocial. We hope the results of this study will help us better understand the implications of being a Duchenne carrier, and possibly apply some of the answers to our understanding of Duchenne in males.
On February 1, 2017, PPMD and FibroGen hosted a webinar to discuss FibroGen’s investigational drug Pamrevlumab (FG-3019), along with FibroGen’s Mission DM
ICYMI, FibroGen joined us earlier this week for a webinar discussion on the company’s investigational drug Pamrevlumab (FG-3019), which is an anti-CTGF monoclonal antibody, along with FibroGen’s Mission DMD program that is currently being conducted to investigate Pamrevlumab in Duchenne. Our speakers provided an overview of the rationale for evaluating Pamrevlumab in Duchenne patients, as well as a description of the recent protocol amendment for this ongoing clinical trial in non-ambulatory boys with Duchenne.
CAMBRIDGE, Mass.--(BUSINESS WIRE)--Jan. 19, 2017-- Catabasis Pharmaceuticals, Inc. (NASDAQ:CATB), a clinical-stage biopharmaceutical company, today announced the publication of Phase 1 data on edasalonexent in adult subjects. Edasalonexent is a potential disease-modifying therapy being developed for Duchenne muscular dystrophy (DMD). The Phase 1 trials demonstrated that edasalonexent (CAT-1004), an oral inhibitor of NF-kB, was safe, well tolerated, and inhibited activated NF-kB in adult subjects and the data are presented in an article titled “A Novel NF-kB Inhibitor, Edasalonexent (CAT-1004), in Development as a Disease-Modifying Treatment for Patients with Duchenne Muscular Dystrophy: Phase 1 Safety, Pharmacokinetics, and Pharmacodynamics in Adult Subjects” in the Journal of Clinical Pharmacology (J Clin Pharmacol. 2017 Jan 11. doi: 10.1002/jcph.842.)
Catabasis Pharmaceuticals, Inc., a clinical-stage biopharmaceutical company, today announced the publication of preclinical data on the edasalonexent program, a potential disease-modifying therapy for Duchenne muscular dystrophy (DMD). The preclinical data demonstrate that edasalonexent (CAT-1004) and an analog, CAT-1041, oral inhibitors of NF-kB, are effective in ameliorating the dystrophic process in two animal models of DMD in an article titled “Disease Modifying Effects of Orally Bioavailable NF-kB Inhibitors in Dystrophin-Deficient Muscle” in JCI Insight (JCI Insight 2016 Dec 22;1(21):e90341).
SOUTH PLAINFIELD, N.J., Dec. 8, 2016 /PRNewswire/-- PTC Therapeutics Inc. (NASDAQ: PTCT), today announced that the company received France's 2016 Prix Galien for Translarna™ (ataluren) for the treatment of nonsense mutation Duchenne muscular dystrophy (nmDMD) in the Medicines "Rare Disease" category. The award recognizes the scientific innovation represented by Translarna's ability to target the underlying cause of nmDMD and the impact the drug is having on patients. The award was presented at the annual gala and ceremony in Paris, France.
PTC Therapeutics, Inc. today announced that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has recommended t…
PTC Therapeutics today announced that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has recommended the renewal of the conditional marketing authorization of Translarna (ataluren) for the treatment of nonsense mutation Duchenne. As a condition of the renewal, PTC is required to conduct an additional long-term post-authorization trial.
While PTC's regulatory engagement with the EMA is separate from the engagement with U.S. regulators at the FDA, today’s decision will enable the continuation of global access to Translarna and powerful data collection to further support PTC's pursuit of an access pathway to Translarna for U.S. patients. PPMD is continuing to work closely with PTC and is pleased that European authorities are recognizing the importance of benefit-risk assessments within product reviews.
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