October 28, 2020

WATCH: Catabasis Phase 3 PolarisDMD Trial Results (Webinar Recording)

We were disheartened to learn earlier this week that the results from the Catabasis Phase 3 trial for edasalonexent (PolarisDMD) did not meet its primary or secondary endpoints. On October 27, 2020, PPMD was joined by…

Learn More

October 26, 2020

Catabasis Announces Top-Line Results for the Phase 3 PolarisDMD Trial of Edasalonexent in Duchenne

We are disheartened to learn today the results from the Catabasis Phase 3 trial for edasalonexent (PolarisDMD) did not meet its primary or secondary endpoints. The unfortunate results from the Catabasis study is yet another…

Learn More

October 26, 2020

Antisense Therapeutics Receives US FDA Orphan Drug Designation for ATL1102 for the Treatment of Duchenne

Antisense Therapeutics announced today that the US Food and Drug Administration (FDA) has granted an Orphan Drug Designation to the ATL1102 for treatment of Duchenne muscular dystrophy. ATL1102 is an antisense drug aimed at reducing…

Learn More

October 23, 2020

WATCH: Connect with the Experts – Delayed Puberty (Endocrine Series)

The body’s endocrine system is made up of glands that produce hormones, which are chemical messengers. Examples are growth hormone (that tells your body when to start and stop growing) and testosterone (the male hormone…

Learn More

October 23, 2020

Solid & Ultragenyx Announce Strategic Collaboration to Develop & Commercialize New Gene Therapies for Duchenne

We are pleased that Solid Biosciences has announced a strategic collaboration and license agreement with Ultragenyx Pharmaceutical. Emil Kaakis, CEO of Ultragenyx, has been focused on rare diseases across his career and has considerable experience…

Learn More

October 20, 2020

WATCH: Connect with the Experts – Bone Health (Endocrine Series)

The body’s endocrine system is made up of glands that produce hormones, which are chemical messengers. Examples are growth hormone (that tells your body when to start and stop growing) and testosterone (the male hormone…

Learn More

October 14, 2020

Publication of Australian Consensus Statement for Powered Wheelchair Standing Devices in Duchenne

As many families know, getting a Powered Wheelchair Standing Device (PWSD) approved and covered by insurance can be a frustrating and exhausting task. Having the same issues in Australia, an international multidisciplinary panel of clinicians,…

Learn More

October 12, 2020

Italfarmaco Receives FDA Rare Pediatric Disease Designation & Completes EPIDYS Phase 3 Trial Enrollment

Italfarmaco has provided an update on the development of Givinostat, sharing that the FDA has granted a Rare Pediatric Disease designation to Givinostat for the treatment of Duchenne, which allows an expedited review process for…

Learn More

October 9, 2020

Connect with the Experts: Endocrine Issues in Duchenne

The body’s endocrine system is made up of glands that produce hormones, which are chemical messengers. Examples are growth hormone (that tells your body when to start and stop growing) and testosterone (the male hormone…

Learn More

October 8, 2020

2020 World Muscle Society: Duchenne Research Updates

Last week the World Muscle Society (WMS) held its 25th Annual Conference. As is often the case during this pandemic, the WMS meeting was held virtually for the first time. WMS provides an opportunity for…

Learn More