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Funding Restrictions

Humane Resources: Funding Restrictions in Rare Rheumatic Diseases

June 20, 2017 John 0

As any rheumatologist knows, most non-medical individuals don’t really know what ‘rheumatology’ is.  Not so much of a bad reputation, as no reputation.  Relatives and […]

Patient Engagement

Rare Disease Clinical Development: The Crucial Need for Patient and Parent Involvement

June 20, 2017 John 0

LuAnn Sabounjian, Head of Clinical Operations & Drug Safety, PROMETRIKA, LLC Rare Disease Clinical Trials February 28th, 2017 was the tenth observance of Rare Disease […]

NLRP12 FCAS2 RARE DISEASE

Living with a rare disease: a husband and father’s perspective

May 24, 2017 John 0

What does rare disease mean to you? To me, rare disease means a mutation called R1016X in a gene called NLRP12 on my son’s and […]

Rare Voices Australia

Australian children living with rare diseases: experiences of diagnosis and perceived consequences of diagnostic delays.

May 15, 2017 John 0

Australian children living with rare diseases: experiences of diagnosis and perceived consequences of diagnostic delays. New paper from Orphanet J Rare Dis. 2017 Apr 11;12(1):68. doi: […]

Orphan Drugs

We must act as England falls behind on access to rare disease medicines

May 14, 2017 John 0

It is crucial that we develop a bespoke process to evaluate ‘orphan’ medicines if we are to eradicate inequality in the system, says Sebastian Stachowiak […]

app-based tool for clinicians

App-based tool for clinicians using facial recognition, AI and genetic big data to improve rare disease diagnosis and treatment

May 14, 2017 John 0

Boston startup FDNA, which makes a suite of apps called Face2Gene that use facial analysis, artificial intelligence and genomic insights in hopes of improving diagnoses […]

Gene Therapy

A New Gene Therapy Cure Just Treated Its First-Ever Patient

May 9, 2017 John 0

Rarefied Cures This March, a child with severe combined immune deficiency (SCID) became the second commercial gene therapy patient ever. The patient started receiving a drug […]

NLRP12.com’s 2017 Scottish Council Manifesto released

May 7, 2017 John 0

For the 2017 Scottish Local Council Elections NLRP12.com have released their manifesto for council candidates throughout Scotland. We had hoped to be able to get […]

Prices of Drugs Rare Disease

Ultra-rare disease drugs: has access in England just got harder?

May 6, 2017 John 0

Story from Andrew McConaghie on PharmaPhorum New rules on market access introduced by NICE from 1 April will affect most new medicines – but gaining […]

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What advice would you give to others?

  • Humane Resources: Funding Restrictions in Rare Rheumatic Diseases
  • Rare Disease Clinical Development: The Crucial Need for Patient and Parent Involvement
  • Living with a rare disease: a husband and father’s perspective
  • Global Genes® to Convene RARE Partnering and Investor Forum to Accelerate Drug Development for Rare Diseases
  • Australian children living with rare diseases: experiences of diagnosis and perceived consequences of diagnostic delays.
  • We must act as England falls behind on access to rare disease medicines
  • App-based tool for clinicians using facial recognition, AI and genetic big data to improve rare disease diagnosis and treatment
  • Three new medicines cleared for use by NHS Scotland
  • NORD Launches Free CME Program to Educate Medical Professionals about Rare Diseases
  • A Nuanced Message: Marketing to the Rare Diseases Community
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