July 14, 2015

Alexion Pharmaceuticals announces Early Access Program

We are pleased to inform you that Alexion Pharmaceuticals is conducting an open-label Early Access Program (EAP) in select countries (U.S., Belgium, Netherlands, France, Spain, and Russia) to provide patients with hypophosphatasia (HPP) access to asfotase alfa.

Potential patients must have onset of signs/symptoms of HPP prior to 18 years of age and a documented diagnosis of HPP based on lab results and HPP-related skeletal abnormalities. For more information visit: ClinicalTrials.gov Identifier: NCT02496689.

July 9, 2015

Hypophosphatasia Drug Approved in Japan

July 9, 2015, The new drug was approved in Japan. Get news.

August 1, 2012

Hypophosphatasia and Social Security (CAL)

Effective August 11, 2012, Hypophosphatasia Perinatal (Lethal) and Infantile Onset Types meet the compassionate allowances (CAL) disability standards.

Social Security has an obligation to provide benefits quickly to applicants whose medical conditions are so serious that their conditions obviously meet disability standards.

Compassionate Allowances (CAL) are a way of quickly identifying diseases and other medical conditions that invariably qualify under the Listing of Impairments based on minimal objective medical information. Compassionate Allowances allow Social Security to target the most obviously disabled individuals for allowances based on objective medical information that they can obtain quickly. Compassionate Allowances is not a separate program from the Social Security Disability Insurance or Supplemental Security Income programs. (this information is from ssa compassionate allowances web page)

For more information go to www.ssa.gov/compassionateallowances