scholarly journals Measuring outcomes in ultra-rare bone diseases: Methodology of the palovarotene fibrodysplasia ossificans progressiva clinical development programme

Bone Reports ◽  
2020 ◽  
Vol 13 ◽  
pp. 100609
Author(s):  
Robert J. Pignolo ◽  
Geneviève Baujat ◽  
Matthew A. Brown ◽  
Carmen De Cunto ◽  
Maja Di Rocco ◽  
...  
Keyword(s):  
Bone Diseases ◽  
Development Programme ◽  
Clinical Development ◽  
Fibrodysplasia Ossificans Progressiva ◽  
Measuring Outcomes ◽  
Rare Bone Diseases

scholarly journals Ligelizumab treatment for severe asthma: learnings from the clinical development programme

2021 ◽  
Vol 10 (3) ◽  
Author(s):  
Jordis Trischler ◽  
Ivan Bottoli ◽  
Reinhold Janocha ◽  
Christoph Heusser ◽  
Xavier Jaumont ◽  
...  
Keyword(s):  
Severe Asthma ◽  
Development Programme ◽  
Clinical Development

Clinical Development Programme for Omeprazole

Digestion ◽  
1990 ◽  
Vol 47 (1) ◽  
pp. 54-58 ◽  
Author(s):  
A. Walan
Keyword(s):  
Development Programme ◽  
Clinical Development

Proceedings of the 2019 Santa Fe Bone Symposium: New Concepts in the Care of Osteoporosis and Rare Bone Diseases

2020 ◽  
Vol 23 (1) ◽  
pp. 1-20 ◽  
Author(s):  
E. Michael Lewiecki ◽  
John P. Bilezikian ◽  
Risa Kagan ◽  
Deborah Krakow ◽  
Michael R. McClung ◽  
...  
Keyword(s):  
Bone Diseases ◽  
Santa Fe ◽  
New Concepts ◽  
Rare Bone Diseases

scholarly journals Providing high-quality care remotely to patients with rare bone diseases during COVID-19 pandemic

2020 ◽  
Vol 15 (1) ◽  
Author(s):  
E. Brizola ◽  
G. Adami ◽  
G. I. Baroncelli ◽  
M. F. Bedeschi ◽  
P. Berardi ◽  
...  
Keyword(s):  
Quality Care ◽  
Bone Diseases ◽  
High Quality ◽  
High Quality Care ◽  
Rare Bone Diseases

The first recombinant FVIII produced in human cells - an update on its clinical development programme

Haemophilia ◽  
2013 ◽  
Vol 20 ◽  
pp. 1-9 ◽  
Author(s):  
L. A. Valentino ◽  
C. Negrier ◽  
G. Kohla ◽  
A. Tiede ◽  
R. Liesner ◽  
...  
Keyword(s):  
Development Programme ◽  
Clinical Development ◽  
Human Cells ◽  
Recombinant Fviii

scholarly journals Skeletal and Metabolic Bone Disorders with special reference to African Americans

2021 ◽  
Vol 3 (1) ◽  
pp. 01-03
Author(s):  
Prabir Mandal ◽  
Noor Islam ◽  
Anita Mandal
Keyword(s):  
Developmental Disorders ◽  
Autosomal Recessive ◽  
Autosomal Dominant ◽  
Bone Diseases ◽  
Living Tissue ◽  
Bone Disorders ◽  
Skeletal Dysplasias ◽  
The Past ◽  
Metabolic Bone ◽  
Rare Bone Diseases

The human skeleton is living tissue that is either growing or being renewed. Our understanding of the pathogenesis of bone disorders has progressed considerably over the past 37 years. A large number of genetic and developmental disorders affect the skeleton. Rare bone diseases account for 5% of all birth defects. The skeletal dysplasias are inherited in an autosomal recessive, autosomal dominant, X-linked recessive, and X-linked dominant, and Y-linked manner.

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