On May 26, NS Pharma, a wholly owned subsidiary of the Japanese company Nippon Shinyaku Co. Ltd., announced positive results from its phase 2 clinical trial of the investigational therapy viltolarsen for treatment of Duchenne muscular dystrophy (DMD) amenable to skipping exon 53. The primary endpoints of the study, assessment of safety and tolerability, were . . .
In April, MDA fielded a survey to ask its community how COVID-19 was impacting their lives. We heard your responses — anxiety, questions, hope — and wanted to know more. In this six-part blog series, Hacking COVID, people from MDA’s community, all living with neuromuscular diseases, shared how they’ve altered their day-to-day lives, how they’ve . . .
On May 15, Pfizer announced positive results from a phase 1b trial assessing treatment with the company’s investigational gene therapy PF-06939926 in ambulatory boys with Duchenne muscular dystrophy (DMD). Preliminary data indicated that the intravenous administration of PF-06939926 was well-tolerated during the infusion period and provided improvements across multiple efficacy-related endpoints at 12 months post-infusion, . . .
On May 22, Danish company Orphazyme announced Fast Track designation from the US Food and Drug Administration (FDA) for development of the investigational drug arimoclomol for the treatment of amyotrophic lateral sclerosis (ALS). Arimoclomol previously received Fast Track status from the FDA for the treatment of Niemann-Pick disease type C (NPC) and sporadic inclusion body . . .
MDA Care Center physicians across the country are working on the frontlines of the novel coronavirus pandemic. Every day, they’re fielding patient questions and finding new ways to practice medicine in a new time. Today, Dr. Susan Apkon, chief of the Department of Rehabilitation and MDA Care Center Director at Children’s Hospital Colorado, shares how . . .
Over the course of the last several months, the US Food and Drug Administration (FDA) has undertaken many actions aimed at slowing the spread of the novel coronavirus and ensuring our first responders have the medical equipment, devices, diagnostic tools, and therapies to treat those with COVID-19. Simultaneously, the FDA has issued guidance on how . . .
Volunteers are a rich part of MDA’s 70-year history. Through volunteer committees, boards, community events, and Summer Camps where kids build confidence and independence, they are tightly woven into MDA’s fabric. This week is National Volunteer Week, and we couldn’t be prouder of or more grateful for the 33,000 volunteers across the country who make . . .
We know families across the country, especially those in the neuromuscular disease (NMD) community, are concerned about the spread of coronavirus and its associated disease, COVID-19, and the impact on their loved ones. In these uncertain times, MDA’s advocacy team will not stop serving and amplifying the voices of those in the NMD community, especially . . .
On March 9, Acceleron Pharma, Inc., announced that a phase 2 clinical trial of its therapy under development, ACE-083, for the treatment of Charcot-Marie-Tooth disease (CMT) did not show statistically significant improvements in functional tests compared to placebo. As a result, Acceleron is discontinuing development of ACE-083 and its clinical trials program for CMT. Delivered by . . .
On March 9, NS Pharma Inc. (a wholly owned US subsidiary of Nippon Shinyaku Co. Ltd.) announced that it has launched an Expanded Access Program in the United States for viltolarsen, its investigational therapy for the treatment of Duchenne muscular dystrophy (DMD) in patients amenable to skipping exon 53. The program is open to all DMD patients . . .