May 12, 2020

NS Pharma Announces First Patient Enrolled in Phase 3 Trial of Viltolarsen in Japan

NS Pharma, a wholly owned subsidiary of Nippon Shinyaku, has enrolled the first patient in a Phase 3 trial of viltolarsen in Japan. PPMD looks forward to continued updates from NS Pharma ahead of their…

Learn More

April 13, 2020

Sarepta Therapeutics Shares COVID-19 Resources

Sarepta Therapeutics has shared a new COVID-19 resource page on its website, along with a new video providing contact phone numbers for those whom may have questions about continuity of access to Sarepta sponsored clinical…

Learn More

April 6, 2020

Audentes Therapeutics Shares Community Update on Duchenne Program & COVID-19

Audentes Therapeutics has shared an update with the community regarding their Duchenne program and COVID-19. Read the Update from Audentes Dear Duchenne Patient Leaders, On behalf of the team at Audentes, we hope that you…

Learn More

March 25, 2020

NS Pharma Announces Marketing Authorization in Japan of VILTEPSO® for the Treatment of Duchenne

Good news out of Japan! NS Pharma, Inc., announced that their parent company, Nippon Shinyaku Co., Ltd., has received approval for viltolarsen in Japan for people with Duchenne amenable to exon 53 skipping therapy. Viltolarsen…

Learn More

March 23, 2020

NS Pharma Shares Community Letter Regarding COVID-19

NS Pharma, Inc. has shared an update with the community responding to questions about how COVID-19 may be affecting ongoing studies of viltolarsen, the recently launched Expanded Access Program (EAP) and review of the viltolarsen…

Learn More

March 19, 2020

Sarepta Therapeutics Shares Community Update on COVID-19

Sarepta Therapeutics has provided the community with an update addressing questions from the community regarding COVID-19, including updates on current clinical trials and accessing approved therapies. Read Sarepta’s Update March 19, 2020 Dear Duchenne and…

Learn More

March 9, 2020

NS Pharma Announces Launch of Expanded Access Program for Viltolarsen

NS Pharma, Inc. has announced the launch of the viltolarsen Expanded Access Program in the United States for patients with Duchenne muscular dystrophy who are amenable to exon 53 skipping therapy and meet other eligibility…

Learn More

February 10, 2020

NS Pharma Announces NDA Accepted for Filing by the FDA for Antisense Oligonucleotide Viltolarsen

PPMD is thrilled to learn from Nippon Shinyaku Co., Ltd. and NS Pharma, Inc. that the FDA has filed a New Drug Application (NDA) for their antisense oligonucleotide drug NS-065/NCNP-01 (viltolarsen). Viltolarsen is a drug…

Learn More

January 15, 2020

WATCH: Update on Investigational Suvodirsen in Duchenne Muscular Dystrophy – Webinar Recording

On December 20, 2019, Parent Project Muscular Dystrophy (PPMD) and World Duchenne Organization (WDO) hosted an informational webinar with the Wave Life Sciences team to discuss their decision to discontinue development of suvodirsen for patients…

Learn More

December 20, 2019

Sarepta Therapeutics Shares Winter 2019 Newsletter

Sarepta has shared the Winter 2019 issue of their newsletter, including updates on current Sarepta programs. Click here to download.

Learn More