Catching Up with the RARE Daily: Giving Tuesday Gratitude & New Stories

December 5, 2014

A big thank you to all who donated on #GivingTuesday to Global Genes! This week’s stories involve new series from our blogger team, op-eds on non-profit investment models and everything new in the word of rare disease.

Regulatory

NICE Recommends PTC’s AADC Gene Therapy

Rare Daily Staff The United Kingdom’s National Institute for Health and Care Excellence, the drug price watchdog, […]

Strategy

PTC and Innozyme CEOs Each Announce Their Retirement

Rare Daily Staff PTC Therapeutics Founding CEO Stuart Peltz said that he is stepping down after 25 […]

Diagnosis

2023 RARE Mental Health Grant Recipients Announced

Global Genes is pleased to announce the Rare Disease Patient Advocacy Organizations receiving 2023 RARE Mental Health […]

podcast

Seeking a Sustainable Business Developing on N-of-1 Therapies

The ability to pinpoint the underlying genetic causes of diseases and rapidly generate genetic medicines to address […]

Diagnosis

Global Genes Team Shares Their Stories on World Rare Disease Day

My daughter Juno was diagnosed with a very rare, severe neurodevelopmental condition called STXBP1-related disorder six years […]

Diagnosis

RARE Global Advocacy Alliance Member Spotlight: Geoffrey Gee

Rare Disease Caregiver Resources

National Caregiver Day in the United States falls on the third Friday of February every year, on […]

Living with a RARE Disease

Finding Me Through This Rare Journey

Patient or Advocate: Brittany Anderson Disease Affiliation: Fibrous Dysplasia, McCune Albright Syndrome, Panhypopituitarism, Adrenal Insufficiency, Trigeminal Neuralgia, […]

Diagnosis