Qure's long-term success hinges on overcoming regulatory and logistical challenges. See why I rate QURE stock a hold.

This particular candidate is being developed to treat patients with Fabry Disease ... Adult Onset Huntington's Disease—This is far more common of the two types, and symptoms from it typically ...

Amicus Therapeutics is to file its oral Fabry disease medicine migalastat with ... requested by the FDA to assess gastrointestinal symptoms is no longer required before the filing.

Developments in genomics are enabling the rise of personalised medicine, with therapies tailored specifically for individuals ...

Also read | Anderson-Fabry Disease: Types, signs and symptoms, diagnosis, treatment of rare genetic disorder that affects organs “Diagnosing Gaucher disease typically involves a combination of ...

An example is screening for spinal muscular atrophy (SMA), which can now be treated before symptoms develop ... for a devastating disease”. Gene therapies for Fabry disease, epilepsy, ALS ...

Fabry's disease is an X-linked disorder caused by ... Patients suffering from the disorder manifest similar symptoms to MPS-I patients, but with differing severities. TKT initiated the pivotal ...

Fabry disease, and Krabbe disease. “They are caused by a genetic defect,” explained Prof Scarpa. “These patients have clinical symptoms in all the different organs of the body: the liver ...