by Peter Ciszewski | Nov 15, 2019
Kristin Smedley, founder of Curing Retinal Blindness Foundation (CRBF) discusses how she used social media and other tools to build her patient advocacy organization and her brand. CRBF is recognized as a leader in the blindness advocacy field, largely due to...
by Peter Ciszewski | Nov 14, 2019
The U.S. Food and Drug Administration has granted Breakthrough Therapy Designation for mavorixafor (X4P-001) to treat adults with WHIM (Warts, Hypogammaglobulinemia, Infections, and Myelokathexis) syndrome. A Breakthrough Therapy Designation is awarded to treatments...
by Peter Ciszewski | Nov 13, 2019
Kevin Freiert, a former Pfizer executive, talks about his podcast ‘Improbable Developments’ that interviews patients and patient advocates in order to learn more about how them and to raise awareness about their disease and/or get a medicine developed for their...
by Peter Ciszewski | Nov 12, 2019
Dennis Jackman, Senior Vice President, Public Affairs at CSL Behring discusses rare disease drug pricing, patient involvement in the drug development process, and drug access. With regard to the latter issue, Jackman said, “if the community and industry,...
by Peter Ciszewski | Nov 11, 2019
New data from a phase III clinical trial may indicate that a drug used in combination therapy for multiple myeloma may be effective as a single-agent therapy. The drug is Ninlaro (ixazomib) is an oral proteasome inhibitor currently approved by the U.S. Food and Drug...
