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RARE News
RARE News
Sickle Cell Disease Association of America promotes Kevin Amado Jr.
By admin
27 March 2024
RARE News
FDA approves nonsteroidal treatment for Duchenne muscular dystrophy
By admin
26 March 2024
RARE News
Rare Disease Day 2024 – Call for greater awareness of X-linked adrenoleukodystrophy and cerebral adrenoleukodystrophy
By admin
20 March 2024
RARE News
Rare disease advocates learn to accelerate therapeutic development at 9th RARE Drug Development Symposium
By admin
19 March 2024
RARE News
Alnylam launches Hereditary ATTR (hATTR) Amyloidosis campaign to help shorten diagnosis time
By admin
19 March 2024
RARE News
New primer highlights latest understanding of rare inherited disease alkaptonuria
By admin
19 March 2024
RARE News
Fortress Biotech and Cyprium Therapeutics announce $4.1 Million grant from NINDS to further development of gene therapy for Menkes Disease
By admin
19 March 2024
RARE News
DEBRA UK asks the British public to BE the difference for EB in 2024
By admin
19 March 2024
RARE News
Launching CamRARE’s ‘The Need for a RARE Patient Passport’ Report for Rare Disease Day
By admin
1 March 2024
RARE News
Coave Therapeutics Receives Grant from the ALS Association to Advance its CTx-TFEB Program as a Potential Treatment for All Forms of ALS
By admin
29 February 2024
RARE News
SMA Europe launches “Care for adults living with SMA in Europe: a benchmarking report.”
By admin
29 February 2024
RARE News
£1.5 million new fund launched to ‘Help the Heart’
By admin
27 February 2024
RARE News
Blueprint Medicines highlights AYVAKIT® (avapritinib) long-term efficacy and safety data and advances in mast cell disease research at 2024 AAAAI annual meeting
By admin
23 February 2024
RARE News
CureDuchenne hosts “Champions in Miami” event on March 9 to help find a cure for Duchenne muscular dystrophy
By admin
23 February 2024
RARE News
CMT Research Foundation partners to advance study of CMT type 1J by Dr Stephan Zuchner
By admin
21 February 2024
RARE News
Patients as Partners in Clinical Research Conference 2024
By admin
19 February 2024
Charity & advocacy
Rare Disease Awareness Day Calendar
By admin
14 February 2024
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