Press Release

AMSTERDAM, November 11 /PRNewswire/ -- Amsterdam Molecular Therapeutics (Euronext: AMT), a leader inthe field of human gene therapy, announced today that it has successfullytreated Duchenne muscular dystrophy (DMD) in an animal model with itsproprietary gene therapy. The proof of concept studies were performed incollaboration with the group of Professor Irene Bozzoni (University of Rome,La Sapienza, Italy) and demonstrated effectiveness in the heart as well as inskeletal muscles. In a previous study, AMT's gene therapy approach was shownto be successful in the treatment of diseased human muscle cells obtainedfrom biopsies of DMD patients. These data establish a robust basis for AMT'stherapeutic approach to DMD.

"We are proud to establish proof of concept with our genetherapy for Duchenne muscular dystrophy, as it is an important new step indeveloping a treatment for this progressive and devastating disease," saidJörn Aldag, Chief Executive Officer of AMT. "In particular, our positiveresults in the heart and respiratory muscles bring hope that we might be ableto prevent the fatal outcome of this disease in the future, potentiallythrough a long-term remedy with a single treatment."

About Duchenne Muscular Dystrophy

Duchenne muscular dystrophy is a severe disease characterizedby progressive muscle degeneration. It affects young children, almostexclusively boys, and leads to paralysis and death in young adulthood. Thedisease is caused by mutations in the dystrophin gene, as a result of whichthe production of functional dystrophin protein, an important structuralcomponent within muscle tissue, is blocked. Currently, there is no treatmentto prevent the fatal outcome of this disease. DMD affects one in 3,500 males,making it the most prevalent of muscular dystrophies.

AMT is developing a gene therapy product for DMD based on'exon skipping' technology which results in bypassing the genetic defect suchthat the functional protein can be formed again. Positive long-termtherapeutic effects of this approach have been demonstrated in animals.

About Amsterdam Molecular Therapeutics

AMT has a unique gene therapy platform that appears tocircumvent many if not all of the obstacles that have prevented gene therapyfrom becoming a mainstay of clinical medicine. Using adeno-associated viral(AAV) vectors as the delivery vehicle of choice for therapeutic genes, thecompany has been able to design and validate what is probably the firststable and scalable AAV production platform. As such, AMT's proprietaryplatform holds tremendous promise for thousands of rare (orphan) diseases,especially diseases that are caused by one faulty gene. Currently, AMT has apipeline with nine products at different stages of research or development.

Certain statements in this press release are "forward-lookingstatements" including those that refer to management's plans and expectationsfor future operations, prospects and financial condition. Words such as"strategy," "expects," "plans," "anticipates," "believes," "will,""continues," "estimates," "intends," "projects," "goals," "targets" and otherwords of similar meaning are intended to identify such forward-lookingstatements. Such statements are based on the current expectations of themanagement of Amsterdam Molecular Therapeutics only. Undue reliance shouldnot be placed on these statements because, by their nature, they are subjectto known and unknown risks and can be affected by factors that are beyond thecontrol of AMT. Actual results could differ materially from currentexpectations due to a number of factors and uncertainties affecting AMT'sbusiness, including, but not limited to, the timely commencement and successof AMT's clinical trials and research endeavors, delays in receiving U.S.Food and Drug Administration or other regulatory approvals (i.e. EMEA, HealthCanada), market acceptance of AMT's products, effectiveness of AMT'smarketing and sales efforts, development of competing therapies and/ortechnologies, the terms of any future strategic alliances, the need foradditional capital, the inability to obtain, or meet, conditions imposed forrequired governmental and regulatory approvals and consents. AMT expresslydisclaims any intent or obligation to update these forward-looking statementsexcept as required by law. For a more detailed description of the riskfactors and uncertainties affecting AMT, refer to the prospectus of AMT'sinitial public offering on June 20, 2007, and AMT's public announcements madefrom time to time.PRN NLD