Duchenne Muscular Dystrophy: Givinostat

Lord Kamall Excerpts

Tuesday 22nd July 2025 Lords Chamber

[Baroness Blake of Leeds (Lab)]

The noble Baroness raises a very interesting point. The drug companies will provide the drugs for free, but it is down to individual trusts to pay the administrative costs. She highlights that one boy in one trust is being supported, while another boy is not. It is a difficult situation; I completely understand that, and my heart goes out to all the young people who suffer from this dreadful disease. It is critical that the trusts work together and look at other practice. It is not for the Government to intervene and force trusts to do as the noble Baroness proposes. However, if they choose to do so, companies can provide funding to enable the clinical administration of their products in advance of the NICE decision. Perhaps the noble Baroness could go back and recommend that as a course of action.

[Lord Kamall (Con)]

My Lords, we heard from the noble Baroness, Lady Kramer, that there is an early access programme for a new treatment of Duchenne muscular dystrophy in the UK. Can the Minister share any intermediate observations or results from the early access programme? As the noble Baroness, Lady Kramer, asked, are there any plans to extend or expand it?