Treatment has been linked to durable, symptom-free, treatment-free remission in several cases of MG.
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After a lukewarm review, the FDA has officially denied the NDA for drisapersen.
Results indicate that IT and IM administration of MSC-NTF cells is safe in patients with ALS and may ultimately provide clinical benefit.
Translarna (ataluren) is for the treatment of nonsense mutation Duchenne muscular dystrophy (nmDMD).
Researchers recommend that follow-up protocols be developed for those with elevated CK but negative DMD gene testing.
The committee cited a lack of consistent clinical trial results as well as concerns over serious adverse events.
Researchers have linked the Jagged1 gene to a much milder form of muscular dystrophy in golden retrievers.
Musculoskeletal pain, especially in the shoulder/arm and leg, is significantly associated with RLS.
The FDA is set to make a decision on the NDA by late February, 2016.
Overall, whole exome sequencing achieved a 45% success rate in difficult-to-diagnose cases of LGMD.
A high fat diet, but low BMI suggest an altered energy metabolism that may contribute to ALS.
The researchers stressed that the results do not indicate a cause-and-effect link between formaldehyde exposure and ALS.
The findings of this study were at odds with previous findings.
The procedure may have benefits for those with stroke, Bell’s palsy, and muscular dystrophy.
The mouse model will allow researchers to effectively test therapeutic compounds.
Genervon has requested accelerated approval from the FDA for its experimental ALS drug, GM604.
The guidelines emphasize a multidisciplinary approach to the diagnosis and treatment of CMDs.
The diagnostic yield was three-fold higher for targeted panel testing vs. single gene testing.
The study was the first to model the disease in a large animal with similar physiology to humans.
Intel worked with Professor Stephen Hawking to develop a better communication platform for people with MNDs.
New diagnosis and treatment recommendations have been created to guide testing for rare muscular dystrophies.
