Rare Diseases

Rare Diseases

Rising to the unique challenges of rare diseasesRising to the unique challenges of rare diseases
Rising to the unique challenges of rare diseases

Aaron Bean at Veeva explores the challenges of rare diseases: With up to 30 million people in EU living with rare diseases, Veeva discusses the challenges facing these rare diseases

Dec 1, 2022 Rare Diseases
The changing landscape of rare disease researchThe changing landscape of rare disease research
The changing landscape of rare disease research

Pharmafile speaks to Chiesi Group: Chiesi Group tells Pharmafile about the challenges facing rare disease research and their hopes for the future of this area

Dec 1, 2022 Rare Diseases
The power of partnership: Collaborating with PAOs for better clinical researchThe power of partnership: Collaborating with PAOs for better clinical research
The power of partnership: Collaborating with PAOs for better clinical research

Patient advocacy organisations can form an effective partnership between clinical trials and patients for more efficient trials

Dec 1, 2022 Rare Diseases
Helping rare diseases emerge from the shadows to take centre stageHelping rare diseases emerge from the shadows to take centre stage
Helping rare diseases emerge from the shadows to take centre stage

The rare disease treatment market has been rapidly changing and receive deserved recognition

Dec 1, 2022 Rare Diseases
The combination treatment challengeThe combination treatment challenge
The combination treatment challenge

Takeda tells Pharmafile about the role of combination treatments in rare diseases, and strategies to make them more accessible

May 31, 2022 Rare Diseases
Rare diseases and exceptional solutionsRare diseases and exceptional solutions
Rare diseases and exceptional solutions

Many advancements have been made within the rare disease field, including gene editing and viral delivery strategies

May 31, 2022 Rare Diseases
Supporting rare neurological disease patients during COVID-19Supporting rare neurological disease patients during COVID-19
Supporting rare neurological disease patients during COVID-19

PHARMAFILE SPEAKS TO The Brain Charity: The Brain Charity explain how they've been providing support to patients with rare diseases throughout the COVID-19 pandemic

Dec 1, 2021 Rare Diseases
Every number has a face: Are you valuing the patient voice?Every number has a face: Are you valuing the patient voice?
Every number has a face: Are you valuing the patient voice?

Patients play an important but often forgotten role in drug development, this article considers the significance of patient experience while developing treatments

Dec 1, 2021 Rare Diseases
Treatable, untreatable, undetermined: Overcoming rare diseaseTreatable, untreatable, undetermined: Overcoming rare disease
Treatable, untreatable, undetermined: Overcoming rare disease

Ipsen tells Pharmafile about the underestimated impacts of rare and ultra-rare diseases

Dec 1, 2021 Rare Diseases
Not so rare diseases: The cumulative burden of underdiagnosisNot so rare diseases: The cumulative burden of underdiagnosis
Not so rare diseases: The cumulative burden of underdiagnosis

A lack of research, funding and representation for rare diseases can lead to under diagnosis, this article looks to the future of rare disease treatments

Dec 1, 2021 Rare Diseases
Rare disease and orphan medicines – Will gover nment policy change the paradigm?Rare disease and orphan medicines – Will gover nment policy change the paradigm?
Rare disease and orphan medicines – Will gover nment policy change the paradigm?

Rare and ultra-rare diseases impact a surprising amount of people, this article explores the field of rare diseases as well as the diagnostic journey many patients take

Dec 1, 2021 Rare Diseases