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rare disease advocates

- PMLiVE

UCB’s Kygevvi receives positive CHMP opinion for thymidine kinase 2 deficiency

TK2d is an extremely rare disease characterised by severe and progressive muscle weakness

- PMLiVE

Sanofi announces positive results for venglustat in type 3 Gaucher disease

There are currently no approved treatments for the neurological symptoms of this disease

- PMLiVE

Havas Life London unveils AI awareness campaign

The campaign highlights the dangers of AI misinformation in rare disease

- PMLiVE

FDA approves first gene therapy for Wiskott-Aldrich syndrome

Symptoms of the rare disease include bleeding, eczema and frequent infections

- PMLiVE

AstraZeneca announces $2bn investment in US manufacturing

The new and expanded facilities will support production of rare disease drug treatments

- PMLiVE

AstraZeneca reports positive results from rare disease trial

HES is a group of rare disorders characterised by persistently elevated levels of white blood cells

- PMLiVE

Medical Misinformation and Sensationalism in the Age of Social Media

In today’s digital age, misinformation spreads faster than ever, often driven by sensational headlines and influencer content. These sources of misinformation often overshadow evidence-based content, creating confusion for both healthcare...

Medscape Education

- PMLiVE

Amgen’s Uplizna approved by FDA as first treatment for rare disease IgG4-RD

Immunoglobulin G4-related disease affects approximately 20,000 people in the US

- PMLiVE

FDA approves Soleno’s Vykat XR as first treatment for hyperphagia in Prader-Willi syndrome

The rare genetic disorder affects an estimated one in every 15,000 newborns in the US

- PMLiVE

Novartis’ Fabhalta granted FDA approval to treat ultra-rare kidney disease C3G

Approximately half of C3G patients progress to kidney failure within ten years of diagnosis

QR codes for prescribing info

Leveraging the ABPI QR Code Update: Enhancing Prescribing Information Through Best Practices

ABPI's recent update permits QR codes in printed pharmaceutical materials for prescribing information. By ensuring visibility, providing clear instructions, linking directly, and keeping content current, companies can enhance accessibility, ensure...

Genetic Digital

- PMLiVE

Novartis shares positive phase 3 results for spinal muscular atrophy gene therapy

The company said it is planning to file regulatory applications for OAV101 IT this year

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