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PTC Therapeutics to Present New Data at the 2022 Muscular Dystrophy Association Clinical & Scientific Conference

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PTC Therapeutics (NASDAQ: PTCT) announced the presentation of three posters on EMFLAZA™ (deflazacort) for Duchenne muscular dystrophy (DMD) at the MDA Clinical & Scientific Conference from March 13-16, 2022, in Nashville, TN. This marks the first presentation of data regarding steroid treatment outcomes in non-ambulatory DMD patients, supporting deflazacort's advantages over prednisone. Key presentations include associations between both steroid treatments and disease progression markers and milestones. A hybrid industry forum on corticosteroid treatment is set for March 15.

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SOUTH PLAINFIELD, N.J., March 11, 2022 /PRNewswire/ -- PTC Therapeutics, Inc. (NASDAQ: PTCT) today announced that the company will present three posters outlining data for EMFLAZA™ (deflazacort) in Duchenne muscular dystrophy (DMD) at the Muscular Dystrophy Association (MDA) Clinical & Scientific Conference being held March 13-16 in Nashville, Tenn. and virtually.

For the first time, PTC will be presenting data on the association between steroid treatment and clinical outcomes among non-ambulatory patients with DMD, expanding the body of evidence that supports the potential cumulative benefit of deflazacort versus prednisone in this patient population. These results have not been previously published and further advance the clinical understanding of steroid treatment in the continuum of DMD care.

Poster presentations include:

  • Poster Title: Associations Between Deflazacort vs Prednisone/prednisolone and Disease Progression Markers in Subgroups of Patients with Duchenne Muscular Dystrophy
    Poster Number: 109

  • Poster Title: Associations Between Daily Deflazacort or Prednisone and Ages at Disease Progression Milestones Among Patients with Duchenne Muscular Dystrophy
    Poster Number: 110

  • Poster Title: Associations Between Steroid Treatment and Clinical Outcomes Among Non-Ambulatory Patients with Duchenne Muscular Dystrophy
    Poster Number: 95

Posters will be viewable in-person throughout the conference. All abstracts are available on the MDA Conference website at https://mdaconference.org/abstract-search.

PTC also will host a hybrid industry forum, "Throughout the Disease Continuum: Corticosteroid Treatment in Duchenne Muscular Dystrophy," on Tuesday, March 15 from 12:00-1:25 p.m. CDT. Those interested in attending in person or virtually can register at http://www.PTCIF2022.com. Participation is limited to licensed U.S. healthcare practitioners only.

About PTC Therapeutics, Inc. 
PTC is a science-driven, global biopharmaceutical company focused on the discovery, development and commercialization of clinically differentiated medicines that provide benefits to patients with rare disorders. PTC's ability to innovate to identify new therapies and to globally commercialize products is the foundation that drives investment in a robust and diversified pipeline of transformative medicines. PTC's mission is to provide access to best-in-class treatments for patients who have little to no treatment options. PTC's strategy is to leverage its strong scientific and clinical expertise and global commercial infrastructure to bring therapies to patients. PTC believes this allows it to maximize value for all its stakeholders. To learn more about PTC, please visit us at www.ptcbio.com and follow us on Instagram, Facebook, Twitter, and LinkedIn.

For More Information:
Investors:
Kylie O'Keefe
+1 (908) 300-0691
kokeefe@ptcbio.com

Media:
Jeanine Clemente
+1 (908) 912-9406
jclemente@ptcbio.com

Forward-Looking Statements
This press release contains forward-looking statements within the meaning of The Private Securities Litigation Reform Act of 1995. All statements contained in this release, other than statements of historic fact, are forward-looking statements, including statements regarding: the future expectations, plans and prospects for PTC, including with respect to the commercialization of its products and product candidates; PTC's strategy, future operations, future financial position, future revenues, projected costs; and the objectives of management. Other forward-looking statements may be identified by the words "guidance", "plan," "anticipate," "believe," "estimate," "expect," "intend," "may," "target," "potential," "will," "would," "could," "should," "continue," and similar expressions.

PTC's actual results, performance or achievements could differ materially from those expressed or implied by forward-looking statements it makes as a result of a variety of risks and uncertainties, including those related to: the outcome of pricing, coverage and reimbursement negotiations with third party payors for PTC's products or product candidates that PTC commercializes or may commercialize in the future; significant business effects, including the effects of industry, market, economic, political or regulatory conditions; changes in tax and other laws, regulations, rates and policies; the eligible patient base and commercial potential of PTC's products and product candidates; PTC's scientific approach and general development progress; and the factors discussed in the "Risk Factors" section of PTC's most recent Annual Report on Form 10-K, as well as any updates to these risk factors filed from time to time in PTC's other filings with the SEC. You are urged to carefully consider all such factors.

As with any pharmaceutical under development, there are significant risks in the development, regulatory approval, and commercialization of new products. There are no guarantees that any product will receive or maintain regulatory approval in any territory or prove to be commercially successful, including Emflaza.

The forward-looking statements contained herein represent PTC's views only as of the date of this press release and PTC does not undertake or plan to update or revise any such forward-looking statements to reflect actual results or changes in plans, prospects, assumptions, estimates or projections, or other circumstances occurring after the date of this press release except as required by law.

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SOURCE PTC Therapeutics, Inc.

FAQ

What is the significance of PTC Therapeutics' presentation at the MDA Conference on March 15, 2022?

PTC Therapeutics' presentation highlights new data on EMFLAZA™ and its benefits in Duchenne muscular dystrophy, particularly focusing on steroid treatment outcomes.

What are the poster titles presented by PTC Therapeutics at the MDA Conference?

The poster titles include 'Associations Between Deflazacort vs Prednisone/prednisolone and Disease Progression Markers,' 'Associations Between Daily Deflazacort or Prednisone and Ages at Disease Progression Milestones,' and 'Associations Between Steroid Treatment and Clinical Outcomes Among Non-Ambulatory Patients with DMD.'

When and where will the MDA Clinical & Scientific Conference take place?

The MDA Clinical & Scientific Conference will be held from March 13-16, 2022, in Nashville, TN, and virtually.

What data is PTC Therapeutics presenting related to DMD at the conference?

PTC will present data on the association between steroid treatment and clinical outcomes among non-ambulatory DMD patients, comparing the effects of deflazacort with prednisone.

How can I attend PTC Therapeutics' hybrid industry forum on corticosteroid treatment?

Interested participants can register for PTC's hybrid industry forum on March 15, 2022, at www.PTCIF2022.com.

PTC Therapeutics, Inc.

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