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New Horizons for Metachromatic Leukodystrophy
7
Mins
March 2025
This article summarises an Orchard Therapeutic-sponsored symposium titled ‘New horizons for metachromatic leukodystrophy – with the advent…
Read more
7
Mins
17 Sep 2020
Digit-
all
: Rare Diseases
Rare diseases are increasingly recognised as a global public health priority.1 Whilst rare diseases have a low prevalence individually, it is estimated…
5
Mins
17 Sep 2020
Superficial Ulcerating Rheumatoid Necrobiosis Associated with Methotrexate Use in a Patient with Rheumatoid Arthritis
Rheumatoid arthritis (RA) is a systemic autoimmune condition prevalent in North America and Northern European populations with an incidence of 1%…
10
Mins
17 Sep 2020
What Makes the Lung Unique – Tissue-Specific Immunity in the Respiratory Tract
As the mediator of gas exchange, the lung is an essential organ for mammalian survival and has thus evolved a diverse and intricate set of defence…
11
Mins
17 Sep 2020
Treatment of Interstitial Cystitis/Bladder Pain Syndrome: A Contemporary Review
Interstitial cystitis/bladder pain syndrome (IC/BPS), is among one of the most common diagnoses that give rise to chronic pelvic pain…
11
Mins
4 Aug 2020
Nutritional Management of Paediatric Crohn’s Disease
For many people with Crohn’s disease (CD), onset occurs in childhood or adolescence. Treatment for CD has moved from predominantly surgical to…
3
Mins
23 Jun 2020
Newborn Screening: Now and in the Future
WHAT is the value of newborn screening (NBS) to the rare diseases community?…
2
Mins
23 Jun 2020
Driving Care Pathways into the Future
ARTIFICIAL intelligence could one day form the backbone of healthcare, but with such a powerful technology looming on our horizon, what exactly does…
3
Mins
23 Jun 2020
Disruptive Innovations in Clinical Research
New trends in clinical research and trial design were discussed, as well as challenges associated with the need to perform innovatively to advance…
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