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. […]

READ MORE
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 […]

READ MORE
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

READ MORE
15 Feb
  • By leilanifera
  • Cause in

Summit

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

READ MORE
15 Feb
  • By leilanifera
  • Cause in

PTC Therapeutics

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

READ MORE
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

READ MORE
15 Feb
  • By leilanifera
  • Cause in

cTAP

Approach: Statistical analysis of natural history studies
Funding Impact: Collaboration amongst biotech/pharma to share data and enrich clinical trial design

READ MORE
15 Feb
  • By leilanifera
  • Cause in

Nationwide Children’s Hospital

Approach: Exon skipping (Duplication exon 2 mutations)
Funding Impact: Advanced pre-clinical IND package enabling clinical trial for Duchenne boys

READ MORE
15 Feb
  • By leilanifera
  • Cause in

Exonics

Approach: Gene editing/CRISPR
Funding Impact: Helped found company and fund pre-clinical safety and efficacy research

READ MORE