Discover how AI-powered tools are transforming rare disease diagnosis, patient care, and clinical decision-making.
How was a new rare disease discovered through Singapore’s Undiagnosed Disease Program? And how will technology allow us to ...
For a child with a rare disease, a doctor’s continuing education can end a years-long diagnostic odyssey and unlock access to ...
Nurses play a vital and multifaceted role in rare disease care, providing consistent patient support, education, and care ...
Montez, Medscape’s Rare Disease Education Lead recently spoke with Dr Lucy McKay, CEO, and Megan Pullein, Strategic Operations Officer from the UK-based charity Medics for Rare Disease (MfRD). We ...
PPi specialises in providing guidance and implementation services for healthcare provision. PPi supports the development of market access strategies as well as the ultimate implementation of such ...
Welcome to Zomi Communications! We deliver excellence in organisational and behavioural change, through an inspirational mix of training, coaching and consultancy. What makes Zomi Communications stand ...
The NHS has treated the first patient with Zolgensma, Novartis’ spinal muscular atrophy (SMA) gene therapy. Five-month-old Arthur Morgan, who was diagnosed with SMA in May, has received the once-off ...
When influence outpaces expertise However, the rise of health content on social media has also fuelled the parallel problem ...
Enzyvant Therapeutics (Enzyvant) and Altavant Sciences (Altavant) have announced a merger to form a biopharmaceutical company focused on accelerating the development of therapies for patients with ...
Roche’s Evrysdi (risdiplam) tablets have been approved by the Medicines and Healthcare products Regulatory Agency (MHRA) to treat spinal muscular atrophy (SMA). The decision applies to patients living ...
Novartis has responded to criticism over its managed access programme for spinal muscular atrophy (SMA) gene therapy Zolgensma, with patient groups accusing the company of exercising an unfair ‘health ...