Author: jacob

What is a primary immunodeficiency? A primary immunodeficiency is an umbrella term for a group of conditions that hinder the body’s ability to fight infection. Sometimes referred to as a primary immune disorder, primary immunodeficiency is a group of over 550 chronic, inborn genetic conditions. Whilst in most circumstances they are genetically inherited, some symptoms…

Rare Disease Day 2026, observed on 28 February, highlights the ongoing need to improve access to timely diagnosis, effective treatment, and appropriate care for people living with rare and ultra rare diseases. Marked annually on the last day of February, the campaign brings together patients, clinicians, researchers, and policymakers to address the systemic challenges that…

Stem cell therapy, cell therapy clinical trials and gene therapy are transforming how complex diseases are studied and treated. From regenerative medicine to gene therapy for cancer, these approaches aim to target disease at a cellular or genetic level rather than simply managing symptoms. We’ll explore how gene therapy works, the role of stem cell…

Amyotrophic lateral sclerosis (ALS) is a progressive neurological condition that affects motor neurons in the brain and spinal cord. These nerve cells control voluntary muscle movement. As ALS progresses, motor neurons deteriorate and die, leading to increasing muscle weakness and loss of physical function. What Is ALS? Motor neurons transmit signals from the brain and…

Chordoma is a very rare cancer, diagnosed in only around one in a million people each year. It develops slowly, close to critical structures in the spine and skull base, meaning treatment can be complex and the path to diagnosis long. With limited therapeutic options and a high risk of recurrence, continued oncology-led clinical research…

 A new study by scientists at University College London and Great Ormond Street Hospital has demonstrated promising results in the treatment of T-cell acute Lymphoblastic Leukaemia (T-ALL), a rare and aggressive blood cancer What the study reveals. This groundbreaking research introduces BE-CAR7, a world-first base-edited Chimeric antigen receptor (CAR) T-cell therapy used in humans. The…

Alpha-1 Antitrypsin Deficiency (AATD) is a hereditary condition that increases the risk of serious lung and liver disease. It occurs when the body produces too little of a protective protein called Alpha-1 Antitrypsin (AAT). Without adequate AAT, lung tissue becomes more susceptible to inflammation and progressive damage, while abnormal protein can build up in the…