Rare Disease Report
Patients & Caregivers


Biopharmaceutical company AbbVie has announced the start of the 2017 Thriving Student Scholarship contest for cystic fibrosis patients.
Like many lipodystrophy patients, she appeared to be very healthy. But the disease is a silent killer.
There are currently 75 Progeria children in clinical trials but with 15 compounds under consideration for trials, the math doesn't work.
At WORLDSymposium, Broomfield et al performed a retrospective review of all Hunter syndrome [mucopolysaccharidosis (MPS) II] patients managed at Royal Manchester Children's Hospital, Manchester, United Kingdom since the commercial introduction of Elaprase (idursulfase).
A recently published review article in Expert Opinion of Orphan Drugs, Drs Spinazzola and Kunkel provide an overview of the leading drugs in development for treating the secondary effects of DMD.
Among the recommendations to reduce risk of SIDS are to have the baby sleep in the parents' bedroom and to keep soft objects away from the infant's sleeping area. Breastfeeding is also recommended
While the sickle cell trait is not rare or life-threatening, studies show that participating in physical activities may put someone diagnosed with the trait at higher risk of cardiovascular problems that can be life threatening.
When examining metabolic syndrome, lifestyle modification involving disciplined dietary control and increased physical activity can become more critical than medical therapy.
The triple drug study found increased bone mineral density, but no other improvement. A new study is underway with lonafarnib with everolimus.
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