Rare Disease Report


Patients with myeloproliferative neoplasms (MPN) with essential thrombocythemia (ET) or polycythemia vera (PV) were found to benefit from hydroxyurea therapy. 
This morning, Pfizer announced that its investigational therapy for the treatment of patients with transthyretin (TTR) cardiomyopathy, tafamidis, received Breakthrough Therapy designation from the US FDA.
New data shows that an increase in the burden of AF is independently associated with a higher risk of ischemic stroke and arterial thromboembolism in patients who have not received anticoagulant medication.
New data revealed by the Tufts Center for the Study of Drug Development concludes that orphan drug development takes 18% longer than the average time required for other new drugs.
Dosing of the first patient in the Phase 2 clinical trial of AG10 in patients with ATTR cardiomyopathy, Eidos Therapeutics, Inc. has announced.
The first patient has been dosed in MyoKardia’s Phase 2 MAVERICK-HCM clinical trial of mavacamten in symptomatic non-obstructive hypertrophic cardiomyopathy patients.
In the ATTR-ACT study, tafamidis exhibited a statistically significant reduction in all-cause mortality and frequency of cardiovascular-related hospitalizations in transthyretin cardiomyopathy patients.
Clinical data from an ongoing Phase 2 study of PBI-4050 shows that the drug provides a clear clinical benefit in patients with Alstrom syndrome.
At the 16th International Symposium on Amyloidosis, Alnylam presented results from the APOLLO Phase 3 study of patisiran, which targets transthyretin in hATTR amyloidosis.
This morning, Alynylam Pharmaceuticals, Inc. has announced the Bridge the Gap initiative in which it will partner with a family affected by hATTR amyloidosis for a new book.

The initiative, called , will allow the family that has lived with the rare disease for generations to share its personal accounts. The book is titled “Living a Rare Life,” and can be found, along with other educational content, at hATTRBridge.com.
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