Meet Jenn

A woman living with cystinosis, a rare metabolic disease that can damage many organs and tissues, particularly the kidneys and eyes.


CYSTARAN (CYSTEAMINE OPHTHALMIC SOLUTION) 0.44% STERILE is a cystine-depleting agent indicated for the treatment of corneal cystine crystal accumulation in patients with cystinosis.


To minimize contaminating the dropper tip and solution, care should be taken not to touch the eyelids or surrounding areas with the dropper tip of the bottle. Keep bottle tightly closed when not in use.
There have been reports of benign intracranial hypertension (or pseudotumor cerebri) associated with oral cysteamine treatment that has resolved with the addition of diuretic therapy. There have also been reports associated with ophthalmic use of cysteamine; however, all of these patients were on concurrent oral cysteamine.
CYSTARAN contains benzalkonium chloride, which may be absorbed by soft contact lenses. Contact lenses should be removed prior to application of solution and may be reinserted 15 minutes following its administration.
CYSTARAN is for topical ophthalmic use only.
The most frequently reported ocular adverse reactions occurring in ≥ 10% of patients were sensitivity to light, redness, and eye pain/irritation, headache and visual field defects.

Please see full prescribing information for contraindications, precautions and adverse reactions.

Meet Ashley

A young woman living with ADA-SCID, a rare, inherited disorder that damages the immune system.


Adagen® (pegademase bovine) Injection is indicated for enzyme replacement therapy for adenosine deaminase (ADA) deficiency in patients with severe combined immunodeficiency disease (SCID) who are not suitable candidates for – or who have failed – bone marrow transplantation.



There is no evidence to support the safety and efficacy of Adagen as preparatory or support therapy for bone marrow transplantation. Since Adagen is administered by intramuscular injection, it should be used with caution in patients with thrombocytopenia and should not be used if thrombocytopenia is severe.



  • Any laboratory or clinical indication of a decrease in potency of Adagen should be reported immediately by telephone to Leadiant Biosciences, Inc. Tel. 1-866-792-5172.
  • Appropriate care to protect immune deficient patients should be maintained until improvement in immune function has been documented. The degree of immune function improvement may vary from patient to patient and, therefore, each patient will require appropriate care consistent with immunologic status.

Laboratory Tests

  • The treatment of SCID associated with ADA deficiency with Adagen should be monitored by measuring plasma ADA activity and red blood cell dATP levels.
  • Once treatment with Adagen has been initiated, a desirable range of plasma ADA activity (trough level before maintenance injection) should be 15-35 µmol/hr/mL and after 2 months of maintenance treatment with Adagen, red cell dATP levels should decrease to a range of ≤ 0.005 to 0.015 µmol/mL.
  • Failure to maintain adequate levels of plasma ADA activity in patients undergoing treatment with Adagen may lead to decline in immune function and increase risk of opportunistic infections.

Drug Interactions

  • There are no known drug interactions with Adagen. However, Vidarabine is a substrate for ADA and 2-deoxycoformycin is a potent inhibitor of ADA.


  • Clinical experience with Adagen has been limited. The following adverse reactions were reported during clinical trials: headache in one patient and pain at the injection site in two patients.
  • The following adverse reactions have been identified during post-approval use of Adagen: hemolytic anemia, auto-immune hemolytic anemia, thrombocythemia, thrombocytopenia, autoimmune thrombocytopenia, lymphomas, injection site erythema, and urticaria.

To report SUSPECTED ADVERSE REACTIONS, contact Leadiant Biosciences, Inc. at 1-888-393-4584 or by email at or contact the FDA at 1-800-FDA-1088 or

Please see full prescribing information for contraindications, precautions and adverse reactions.