rare disease

Rare Disease Day 2021

The goal of Rare Disease Day is to raise awareness amongst the general public and decision-makers about rare diseases and their impact on...

AVROBIO Announces 100% Kidney Substrate Reduction at 12 Months Post-Gene Therapy in...

Children’s Friend Inc. is studying the experiences in and barriers to accessing mental health services for families. For the...

November 17, 2020 – AVROBIO announced that data suggests functional and clinical improvements for the first cystinosis patient at...

Learn more about gene therapy as it relates to the cystinosis community. Dr. Paul Grimm from from Stanford University...

Let’s have an open discussion on mental health and living with cystinosis. Everyone within the community is invited! Licensed...

Join hosts from the Adult Leadership Advisory Board (ALAB) for the Cystinosis Sessions video conference series – LIVE- on...

In April, the Cystinosis Network Europe adapted their international conference to the restrictions set forth by COVID-19. The result...

Learn more about the fundamentals of cystinosis during our Cystinosis Basics video meetup. Dr. Larry Greenbaum, Emory School of...

Beginning September 15, 2020, Recordati Rare Diseases announced the availability of CYSTADROPS across the United States. CYSTADROPS are a...