Latest news

• Summit plunges after Duchenne disappointment

  These include the use of CRISPR/Cas9, myostatin Inhibitior candidates from Pfizer and BMS, and Santhera which is developing  Idebenone (Raxone).

• CHMP hands Santhera second ‘no’ in Duchenne muscular dystrophy

  European regulatory advisers have refused to shift their negative position on Santhera’s Duchenne muscular dystrophy (DMD) drug Raxone (idebenone). ... It’s the second such knock-back for Raxone’s licence extension and shares in the Swiss firm

• Summit takes clinical trial participant search online

  Hopes to broaden reach of its Duchenne muscular dystrophy study

• Santhera facing long delay for muscular dystrophy drug in US

  FDA rejects accelerated marketing application for Raxone to await phase II trial results. ... Santhera is facing a considerable delay in the US before it can get approval for Duchenne muscular dystrophy (DMD) candidate Raxone.

• BioMarin gives up on muscular dystrophy drug

  There has been some good news for European DMD patients this week however after Santhera announced it had filed for approval of Raxone (idebenone) for the treatment of DMD.