Chicago-based biotechnology company AveXis has announced encouraging results from its completed phase 1 gene therapy trial to test the investigational therapy AVXS-101 in infants with type 1 SMA.
AB Science has reported encouraging top line trial results from its completed phase 2/3 study in Europe of the experimental drug masitinib in ALS. Additional testing is likely required, but if successful, masitinib could one day become an approved therapy for ALS.
Investigators are seeking participants for an ongoing phase 2 clinical trial, sponsored by Cytokinetics, to test the investigational drug CK-2127107 in people with types 2, 3 or 4 SMA.
Researchers are looking for people with ALS and healthy volunteers to participate in the Microbiome Assessment in People with ALS (MAP ALS) study.
Excited to try MDA’s new web-based Clinical Trials Finder Tool powered by Antidote? Here are some questions we have received and answers that should make using the tool as seamless as possible.
Researchers are looking for people to participate in CReATe Consortium’s PGB study to help learn about ALS and related diseases.
Researchers are looking for 220 people with ALS and 30 people who do not have ALS from around the country to participate in the ALS At HOME study.
Vamorolone (formerly VBP15), under development by ReveraGen BioPharma, has received FDA Fast Track designation for the treatment of DMD.
Akashi Therapeutics announced that the FDA has cleared the way for the company to resume clinical development of HT-100 in boys with DMD.
National Ambassador Joe Akmakjian recently attended the 2017 MDA Scientific Conference. He writes, “I found the conference to be very hopeful and exciting experience. It was an honor to meet the people who have made a career out of saving my life and the lives of the other individuals and families. Everyone connected to MDA should be excited to know that these researchers are actively sharing ideas and discussing ways to solve problems and find the solutions we’re all looking for.”
