Mar. 14 2025
Source Page: Sebetralstat for treating acute attacks of hereditary angioedema in people aged 12 and over ID6284Found: Background Hereditary angioedema (HAE) is a rare genetic disorder, associated with a deficiency or
Mentions:
1: Kevin Bonavia (Lab - Stevenage) Like many unpaid carers across Stevenage, Norman has been put through absolute hell by the previous Tory - Speech Link
2: Mark Ferguson (Lab - Gateshead Central and Whickham) Usher syndrome is a rare inherited disease that can lead to both deafness and blindness. - Speech Link
3: Andrew Gwynne (LAB - Gorton and Denton) The UK rare diseases framework aims to improve the lives of people living with all rare diseases. - Speech Link
Asked by: Lord Hay of Ballyore (Democratic Unionist Party - Life peer)
Question to the Department of Health and Social Care:
To ask His Majesty's Government what provisions are in place to support the families and caregivers of individuals with rare diseases.
Answered by Lord Markham - Shadow Minister (Science, Innovation and Technology)
Each year since 2021, we have published an England Rare Diseases Action Plan. On 29 February 2024, we published the third England Rare Diseases Action Plan. In this plan we recognised that significant challenges exist with access to mental health and psychological support for people living with rare conditions, as well as their families and carers. During this year we have taken steps to further understand the challenges faced through a workshop, and have begun to address these needs by developing resources to better equip the workforce to provide support.
The enormous contribution of unpaid carers, including those caring for individuals with rare diseases, is reflected throughout the Next Steps to Put People at the Heart of Care, published in April 2023. Furthermore, the Better Care Fund in 2023/24 includes £327 million for carers support, including short breaks and respite services for carers. This also funds advice and support to carers, and a small number of additional local authority duties. The Accelerating Reform Fund also provides support for unpaid carers. The Government is developing a new survey of unpaid carers which will capture the wide range of experiences, circumstances, and needs of unpaid carers across England. Through the National Institute for Health and Care Research, an evaluation of the support provided to unpaid carers funded through the Better Care Fund has been commissioned, which will improve the understanding of what support works best for unpaid carers.
Apr. 02 2025
Source Page: Olipudase alfa for treating acid sphingomyelinase deficiency (Niemann–Pick disease) type AB and type BFound: There is also a significant impact on the quality of life of carers and siblings of people with ASMD
Feb. 06 2025
Source Page: Olipudase alfa for treating acid sphingomyelinase deficiency (Niemann–Pick disease) type AB and type BFound: decision should be made jointly by the clinician, the child or young person, and their parents or carers
Feb. 06 2025
Source Page: Olipudase alfa for treating acid sphingomyelinase deficiency (Niemann–Pick disease) type AB and type BFound: 0.080 (children with activity limitation) Adults -0.010 (overall utility from review of chronic diseases
Mentions:
1: Carolyn Thomas (Welsh Labour - None) stable and long-lasting than the carbon in trees, which are vulnerable to forest fires, pests and diseases - Speech Link
2: Rhys ab Owen (Independent Member - None) I asked the carers to play the song 'Yfory'—'Tomorrow'. - Speech Link
Oral Evidence Nov. 26 2024
Inquiry: Proposals for backbench debatesFound: It is one of those rare diseases.
Nov. 26 2024
Source Page: Ganaxolone for treating seizures caused by CDKL5 deficiency disorder in people 2 years and overFound: seizures caused by CDKL5 deficiency disorder in people 2 years and over (ID3988) CDD is an ultra-rare
Oral Evidence Nov. 19 2024
Inquiry: Proposals for backbench debatesFound: It is one of those rare diseases.