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hypoparathyroidism

- 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

EMA reaffirms guidance on paracetamol use during pregnancy

The guidance follows recent US comments about a possible link to autism

- PMLiVE

Johnson & Johnson seeks first EMA approval for icotrokinra for plaque psoriasis

Icotrokinra is a first-in-class, once-daily tablet for moderate-to-severe plaque psoriasis

- PMLiVE

EMA orphan drug designation given to ReAlta for graft-versus-host disease therapy

This follows preliminary results from the company's ongoing phase 2 trial

- 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

Organon/Henlius announce EMA validation for Perjeta biosimilar HLX11

The reference medicine holds approvals for multiple HER2-positive breast cancer indications

- 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

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