An upcoming study will evaluate the effects of trunk-oriented exercises compared to whole-body vibration as therapeutic options for patients with Duchenne muscular dystrophy (DMD). Researchers will soon initiate the recruiting process for this interventional study. The clinical trial will include approximately 30 participants previously diagnosed with DMD with randomized allocation and parallel assignment. Read more…
Gamida Cell is gearing up to begin sales of omidubicel from Israel to US hospitals for use in stem cell transplants, pending FDA approval of the therapy.
Larry Luxner, senior correspondent for Rare Disease Advisor, interviews Abigail Jenkins, president and CEO of Gamida Cell, on the company’s development of omidubicel as an alternative to umbilical cord blood in stem cell transplants for myelofibrosis and other diseases. Read the full story here: Omidubicel Shows Promise as Replacement for Umbilical Cord Blood in Stem…
A new phase 1/2 clinical trial, EXPLORE44™ (NCT05670730), to investigate the use of AOC 1044 in healthy adult volunteers and patients with Duchenne muscular dystrophy (DMD) is now recruiting participants at a study location in San Antonio, Texas. The trial, sponsored by Avidity Biosciences Inc, will enroll patients during 2 different parts of the trial.…
There is a good correlation between lung function and the function of the skeletal muscle in the arms and legs in patients with Duchenne muscular dystrophy (DMD), found a new study that appeared in the journal Pediatric Pulmonology. This is the first study that explores the link between functional measures assessing skeletal muscle disease and…
Friedreich ataxia (FA) is a progressive, inherited, neurodegenerative movement disorder characterized by problems with balance and coordination, muscle weakness, spasticity, difficulty walking, scoliosis, dysarthria, and hearing and vision impairment. These symptoms are caused by degenerative changes in the sensory nerve fibers as they enter the spinal cord in the dorsal root ganglia.1,2 Friedreich ataxia is…
Friedreich ataxia (FA) is a rare, inherited, neurodegenerative condition affecting the brain, spinal cord, and peripheral nerves. Demyelination and atrophy of these nerves cause progressive deterioration of muscular coordination and strength, sensory impairments, and decline in independent mobility.1 Mutations in the FXN gene are the primary underlying cause of FA. These mutations result in the…
Friedreich ataxia (FA) is a neurological condition with autosomal recessive inheritance, characterized by the progressive failure of coordination of the extremities and trunk, spasticity, absence of lower extremity reflexes, dysarthria, and impaired proprioception, vision, and hearing. Although very rare, FA is the most common inherited form of progressive ataxia, occurring in approximately 1 in every…
Friedreich ataxia (FA), the most common inherited form of ataxia in Europe, is a rare, progressive neurodegenerative disorder that primarily affects the nervous system and heart.1 Characteristics signs of FA are progressive gait and limb ataxia, lower limb areflexia, dysarthria, leg muscle weakness, and a positive Babinski sign (extensor plantar response). The disease also manifests…
Friedreich ataxia (FA) is the most common autosomal recessive form of ataxia.1 The disease occurs due to an unstable guanine-adenine-adenine (GAA) trinucleotide repeat expansion (more than 120 repeats) in the first intron of the frataxin (FXN) gene on chromosome 9 in both alleles.2 Progressive gait and limb ataxia, the absence of lower limb reflexes, abnormal…
