07 Sep
  • By Nic Williams
  • Cause in

CureDuchenne Ventures Commits $500,000 to 4D Molecular Therapeutics to Advance Next-Generation AAV Delivery of Gene Therapy

NEWPORT BEACH, Calif. – (BUSINESS WIRE) – CureDuchenne, a national venture philanthropy nonprofit focused on finding a cure for Duchenne muscular dystrophy (DMD), announced today a $500,000 investment into 4D Molecular Therapeutics (4DMT), a world-leader in Therapeutic Vector Evolution for adeno-associated virus (AAV) gene therapy vector discovery and product development, in an effort to advance […]

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30 Aug
  • By Nic Williams
  • Cause in

Crispr Halted Muscular Dystrophy In Dogs. Someday, It Might Cure Humans.

Dear Friends, Today, our partner Dr. Eric Olson and his team at Exonics, a company CureDuchenne helped form, published data in the online journal, Science that shows an increase in dystrophin restoration of 92% in the heart of the canine’s. As the only Duchenne nonprofit organization to support Dr. Eric Olson’s work through Exonics, and […]

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17 Aug
  • By CureDuchenne User
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Santhera Pharmaceuticals now enrolling for SIDEROS Clinical Trial

Dear Friends, We are pleased to provide you with detailed information from Santhera Pharmaceuticals about an ongoing clinical trial that your son may be able to participate in. Beyond investing in early scientific research to accelerate a pathway to a cure for Duchenne, CureDuchenne is committed to providing the Duchenne community with education, help and […]

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16 Jul
  • By Nic Williams
  • Cause in

PTC Now Enrolling for Ataluren Clinical Trial

PTC Therapeutics Update on Ataluren Study 041 PTC Therapeutics is enrolling boys and young men with a nonsense mutation in a clinical trial for Ataluren.  To ensure that participants who enroll in Study 041 get the most benefit, and to ensure their safety, there are strict criteria around who is able to enter the study. […]

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28 Jun
  • By Nic Williams
  • Cause in

Report from the 2018 New Direction in Biology and Disease of Skeletal Muscle Conference

The third day of the conference focused on stem cells, new small molecule screens, poster sessions and clinical trial updates. Hirofumi Komaki (National Center of Neurology and Psychiatry, Japan) presented results of a Japanese Phase I/II dose-finding clinical study with NS-065, an exon-53 skipping drug (NS Pharma) for the treatment of Duchenne. The drug was […]

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15 Feb
  • By leilanifera
  • Cause in

TRiNDS

Approach: Neuromuscular focused Contract Research Organization
Funding Impact: Clinical trial support for Biotech/Pharma from a patient-centric perspective

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15 Feb
  • By leilanifera
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Summit

Approach: Utrophin upregulation
Funding Impact: Enabled initiation of Phase 2 clinical trial

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15 Feb
  • By leilanifera
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Sarepta Therapeutics

Approach: Exon skipping
Funding Impact: First drug approved by FDA to treat Duchenne

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15 Feb
  • By leilanifera
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PTC Therapeutics

Approach: Stop codon read-through
Funding Impact: Conditional approval of Translanrna in EU

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15 Feb
  • By leilanifera
  • Cause in

Prosensa/BioMarin

Approach: Exon skipping
Funding Impact: Helped support clinical development of exon skipping drugs
Successful Exit: Prosensa was acquired by BioMarin in 2015

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