about FDA approves first therapy for rare inherited form of rickets, x-linked hypophosphatemia (aphanet.org)
And a third, different kind of gene therapy from Spark Therapeutics — to treat a rare inherited form of blindness — is likely to be approved by the end of 2017, too, making this year a landmark for treatments that manipulate the body's own biological mechanisms in novel new ways to fight deadly diseases. (fortune.com)
Glitches in the presenilin - 1 gene can cause the rare inherited form of Alzheimer's that strikes at a younger age. (scientificamerican.com)