News

Summit Therapeutics has opened enrollment for an additional group in its ongoing PhaseOut DMD trial to include patients with Duchenne muscular dystrophy (DMD) who participated in the previous Phase 1 clinical trials for the investigational therapy ezutromid (SMT C1100) but who, at the time of enrollment, failed to meet the enrollment…

At a time of unprecedented polarization in Congress, two U.S. lawmakers — one Republican, one Democrat — are stressing the urgency of working across the aisle to help the estimated 30 million Americans with rare diseases. Rep. Leonard Lance (R-New Jersey) and Sen. Amy Klobuchar (D-Minnesota) spoke to more…

Plasticity Brain Centers has become an authorized care provider for children and young adults with muscular dystrophy and other rare disorders who receive Gardiner Scholarships from the State of Florida. In addition to those with rare diseases, the scholarships allow youngsters with developmental disorders and other conditions to attend a private school or…

A Phase 2 clinical trial of ATL1102, Antisense Therapeutics’ investigational therapy for Duchenne muscular dystrophy (DMD), recently received authorization to proceed from the Royal Children’s Hospital (RCH), Melbourne Human Research Ethics Committee. ATL1102 inhibits the protein CD49d, which is required for the normal function of the VLA-4 (very late…

Retrophin and the U.S. subsidiary of Britain’s Horizon Pharma will each donate $3 million over a six-year period to the Rare Disease Institute (RDI) at Children’s National Health System in Washington, D.C., helping it to strengthen care available and expand as a “center of excellence” for rare…

A new interim analysis of the PhaseOut DMD clinical trial shows that ezutromid (SMT C1100) significantly decreased muscle inflammation in patients with Duchenne muscular dystrophy (DMD), Summit Therapeutics announced. Ezutromid is called a utrophin modulator because it is able to trigger utrophin production in people who lack dystrophin,…

In recognition of Rare Disease Day 2018, Bionews Services — which publishes this website — will attend and report on three relevant conferences in the U.S. dealing with policies and programs of importance to patients and their families. The three are among 50 events in 32 states…

A stem cell therapy improved the heart function, walking capacity and survival of mice with Duchenne muscular dystrophy, a study reports. The treatment involves what scientists call cardiosphere-derived cells, or CDCs. They are the research equivalent of the stem cell therapy known as CAP-1002 that Capricor Therapeutics is developing to treat…

The investigational treatment Raxone (idebenone) could become available to qualifying Duchenne muscular dystrophy (DMD) patients in the U.S. with respiratory decline following the start of an expanded access program (EAP), Clinigen and Santhera Pharmaceuticals announced. BreatheDMD is an EAP authorized by the U.S. Food and Drug Administration…

A rock-painting contest in Las Vegas. A fashion show in New York. A 7,000-meter race around the Washington Monument that’ll coincide with a similar #Racefor7 event in Bengaluru and Mumbai, India. From Athens to Atlanta, from San Diego to Sydney, people across the globe will mark World Rare Disease…