FDA Grants Conditional Approval For PTC Therapeutics' Gene Therapy As First Ever With Direct Administration In Brain [Yahoo! Finance]
PTC Therapeutics, Inc. (PTCT)
Last ptc therapeutics, inc. earnings: 4/30 04:01 pm
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Source: Yahoo! Finance
The gene therapy, which will be marketed in the United States with the brand name Kebilidi (eladocagene exuparvovec-tneq), is indicated for the treatment of children and adults with aromatic L-amino acid decarboxylase (AADC) deficiency, including the full spectrum of disease severity. Launch preparations are well underway, with centers already identified and surgeons trained in the procedure to deliver the gene therapy. Also Read: PTC Therapeutics Says FDA Lifts Clinical Hold On Investigational Study In Patients With Rare Weak Nerve Cell Disorder AADC deficiency is a rare genetic disorder that results in the inability to synthesize dopamine, a neurotransmitter essential for motor function. Affected individuals may experience symptoms such as delays in gross motor function (head control, sitting, standing, and walking), hypotonia (weak muscle tone), and developmental and cognitive delays. Kebilidi is a gene replacement therapy that is directly administered to the putamen of
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PTCT
News
- InnovationRx: Trump Picks RFK, Jr. And Dr. Oz For Key Healthcare Roles [Forbes]Forbes
- PTC Therapeutics, Inc. (NASDAQ: PTCT) had its price target raised by analysts at JPMorgan Chase & Co. from $51.00 to $62.00. They now have an "overweight" rating on the stock.MarketBeat
- PTC Therapeutics, Inc. (NASDAQ: PTCT) was downgraded by analysts at StockNews.com from a "buy" rating to a "hold" rating.MarketBeat
- Child Thriving After Becoming World's Youngest to Receive Gene Therapy for AADC Deficiency [Yahoo! Finance]Yahoo! Finance
- PTC Therapeutics Announces FDA Approval of AADC Deficiency Gene Therapy [Yahoo! Finance]Yahoo! Finance
PTCT
Earnings
- 11/7/24 - Beat
PTCT
Sec Filings
- 11/14/24 - Form SC
- 11/8/24 - Form SC
- 11/7/24 - Form 10-Q
- PTCT's page on the SEC website