On June 2, ReveraGen BioPharma announced the completion of two-and-a-half years of vamorolone treatment in 41 boys with Duchenne muscular dystrophy (DMD). The long-term data from this study indicated that daily oral administration of vamorolone, even at its highest tested dose, was safe and well tolerated with no serious adverse events. Preliminary data have shown . . .
MDA Care Center providers 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, Jana Unislawski, a clinical case worker and coordinator of the Memory Disorder Clinic at University of Florida Health, shares how . . .
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 . . .
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 . . .
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 . . .
Researchers at Virginia Commonwealth University are seeking participants living with limb-girdle muscular dystrophy type 2I (LGMD2I) to participate in a natural history study. This study aims to better understand disease course so researchers will be able to determine clinically meaningful outcome measures for use in future clinical trials, including the utility of dystroglycan as a biomarker. This two-year . . .
On Feb. 7, NS Pharma Inc. (a wholly owned US subsidiary of Nippon Shinyaku Co. Ltd.), announced that the US Food and Drug Administration (FDA) accepted its New Drug Application (NDA) seeking approval under Priority Review for viltolarsen, an investigational therapy for the treatment of Duchenne muscular dystrophy (DMD) in patients amenable to skipping exon 53. Priority . . .