News Release Details
Rocket Pharmaceuticals Sponsors 3rd Annual PKD Patients’ Forum
May 29, 2019
|
“At Rocket, we are making significant progress towards developing
transformative gene therapies for patients living with devastating, rare
diseases, including PKD,” said
“We are pleased to sponsor the 3rd Annual PKD Patients’ Forum to discuss PKD diagnosis and present and future treatments, including the forthcoming gene therapy clinical trial for PKD,” said Dr. Segovia. “Internationally recognized PKD experts, as well as clinicians in close contact with the patients are attending today’s Forum. Our objective is to educate families and patients and discuss their physical, psychological and social needs.”
Dr. Segovia continued, “Patients with severe PKD suffer greatly as the only treatment options currently available to them are splenectomy and lifelong red blood cell transfusions. Unfortunately, these treatment options are far from optimal and are associated with considerable side effects. The experts at this event have devoted countless years in research and are an instrumental resource to patients and their families.”
Sponsoring PKD Day is part of Rocket's commitment to promoting rare
disease education and awareness. Additional recent initiatives include a
Rocket-sponsored Rare Disease Day educational event on
About Pyruvate Kinase Deficiency
Pyruvate kinase deficiency (PKD) is a rare, monogenic red blood cell
disorder resulting from a mutation in the PKLR gene encoding for
the pyruvate kinase enzyme, a key component of the red blood cell
glycolytic pathway. Mutations in the PKLR gene result in
increased red cell destruction and the disorder ranges from mild to
life-threatening anemia. PKD has an estimated prevalence of 3,000 to
8,000 patients in
About
Rocket Cautionary Statement Regarding Forward-Looking Statements
Various statements in this release concerning Rocket's future
expectations, plans and prospects, including without limitation,
Rocket's expectations regarding the safety, effectiveness and timing of
product candidates that Rocket may develop, to treat Fanconi Anemia
(FA), Leukocyte Adhesion Deficiency-I (LAD-I), Pyruvate Kinase
Deficiency (PKD), Infantile Malignant Osteopetrosis (IMO) and Danon
disease, and the safety, effectiveness and timing of related
pre-clinical studies and clinical trials, may constitute forward-looking
statements for the purposes of the safe harbor provisions under the
Private Securities Litigation Reform Act of 1995 and other federal
securities laws and are subject to substantial risks, uncertainties and
assumptions. You should not place reliance on these forward-looking
statements, which often include words such as "believe," "expect,"
"anticipate," "intend," "plan," "will give," "estimate," "seek," "will,"
"may," "suggest" or similar terms, variations of such terms or the
negative of those terms. Although Rocket believes that the expectations
reflected in the forward-looking statements are reasonable, Rocket
cannot guarantee such outcomes. Actual results may differ materially
from those indicated by these forward-looking statements as a result of
various important factors, including, without limitation, Rocket's
ability to successfully demonstrate the efficacy and safety of such
products and pre-clinical studies and clinical trials, its gene therapy
programs, the preclinical and clinical results for its product
candidates, which may not support further development and marketing
approval, the potential advantages of Rocket's product candidates,
actions of regulatory agencies, which may affect the initiation, timing
and progress of pre-clinical studies and clinical trials of its product
candidates, Rocket's and its licensors ability to obtain, maintain and
protect its and their respective intellectual property, the timing, cost
or other aspects of a potential commercial launch of Rocket's product
candidates, Rocket's ability to manage operating expenses, Rocket's
ability to obtain additional funding to support its business activities
and establish and maintain strategic business alliances and new business
initiatives, Rocket's dependence on third parties for development,
manufacture, marketing, sales and distribution of product candidates,
the outcome of litigation, and unexpected expenditures, as well as those
risks more fully discussed in the section entitled "Risk Factors" in
Rocket's Annual Report on Form 10-K for the year ended
View source version on businesswire.com: https://www.businesswire.com/news/home/20190529005590/en/
Source:
Claudine Prowse, Ph.D.
SVP, Strategy & Corporate Development
Rocket
Pharma, Inc.
The Empire State Building, Suite 7530
New York,
NY 10118
www.rocketpharma.com
investors@rocketpharma.com