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BioWorld - Wednesday, July 6, 2022
Home » Topics » Musculoskeletal, BioWorld

Musculoskeletal, BioWorld
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Ipsen shares move higher after US FDA refile for ultra-rare disease drug palovarotene

June 29, 2022
By Richard Staines
No Comments

Shares in Ipsen SA edged higher June 29 after the firm announced it had refiled its palovarotene NDA with the U.S. FDA for the ultra-rare disease fibrodysplasia ossificans progressiva. The regulator granted a six-month priority review for the drug, which was once written off and had its FDA filing pulled in 2021 after officials asked for further analyses and data.


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Young boy standing with walker

Italfarmaco’s givinostat hits phase III endpoint in DMD

June 27, 2022
By Cormac Sheridan
No Comments
Italfarmaco SpA will seek meetings with the U.S. FDA and the European Medicines Agency to discuss filing requirements for givinostat in Duchenne muscular dystrophy (DMD) on the strength of data from a phase III trial in which those on the drug exhibited a slower decline in their ability to climb four stairs than those on placebo.
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Give ITT a try: PTC heads to FDA (again) with Translarna data in DMD

June 21, 2022
By Randy Osborne
No Comments
After an up-and-down day – mostly up, toward the end – during which the phrase “totality of the data” got air time aplenty, shares of PTC Therapeutics Inc. (NASDAQ:PTCT) closed at $34.07, a rise of $5.66, or almost 20%, on word of top-line data from Study 041 with Translarna (ataluren) in nonsense mutation Duchenne muscular dystrophy (DMD).
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Mast cell releasing histamine during allergic response

EHA 2022: Best in class? Cogent soars on early data in mastocytosis

June 10, 2022
By Jennifer Boggs
No Comments
Analysts have already started tagging Cogent Biosciences Inc.’s bezuclastinib as potentially best in class, after the company presented impressive, though early stage, data at the European Hematology Association Congress in Vienna demonstrating promising efficacy and a possibly differentiating safety profile for the selective KIT D816V inhibitor in advanced systemic mastocytosis.
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Businesswoman pressing dollar sign on touchscreen

Code raises a series A to develop Duchenne and diabetes therapies

June 7, 2022
By Lee Landenberger
No Comments
Code Biotherapeutics Inc. has raised an upsized and oversubscribed series A financing to develop programs for treating rare and genetic diseases that include Duchenne muscular dystrophy and type 1 diabetes.
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Walking with assistance
Newco news

Mitorx tackling impaired sulphide signaling in degenerative disease

April 26, 2022
By Nuala Moran
No Comments
Mitorx Therapeutics Ltd. is poised to develop small molecules that reverse impaired sulphide signaling underlying degenerative diseases ranging from Duchenne muscular dystrophy to Alzheimer’s disease. The company was formed some time ago as a spinout from Exeter University, where the founding scientist Matt Whiteman is professor of experimental therapeutics. It is showing its colors for the first time after closing a seed funding round.
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Rejuvenate gains clearance for ‘groundbreaking’ sarcopenia trial

March 30, 2022
By Nuala Moran
No Comments
In a first of its kind phase I trial, Rejuvenate Biomed NV has secured ethical clearance to induce acute sarcopenia in 42 healthy subjects, aged 65 to 75 years old. That will be achieved by placing a cast on one leg to induce sarcopenia through muscle disuse. The volunteers in the double-blind trial will then receive either RJx-01, an oral small molecule for treating sarcopenia, or placebo.
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Closeup of wheelchair

Bridgebio advancing LGMD2i program with positive phase II readout

March 14, 2022
By Jennifer Boggs
No Comments
Discussions with regulators on a proposed phase III trial design are up next for Bridgebio Pharma Inc., which reported positive phase II data for BBP-418 (ribitol) in patients with limb-girdle muscular dystrophy type 2 (LGMD2i), the first of several clinical readouts expected in 2022, as the Palo Alto, Calif-based company looks to regain its footing after disappointing top-line data for its phase III program in transthyretin amyloid cardiomyopathy leveled the stock late last year.
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Chip Wilson, Solve FSHD

Making lemonade out of Lululemon: Founder funds research into rare muscular dystrophy

March 11, 2022
By Lee Landenberger
No Comments
Lululemon founder Chip Wilson has chipped in $100 million to prime the pump for finding a cure to the rare form of muscular dystrophy that has hampered him for the past 35 years. He’s got more than a monetary stake in the donation. The 67-year-old entrepreneur suffers from facioscapulohumeral muscular dystrophy type 2 (FSHD) and just launched Solve FSHD to find a cure.
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Splicebio raises record series A to tackle AAV packing problem

Feb. 16, 2022
By Nuala Moran
No Comments
LONDON – In the largest-ever series A for a Spanish biotech, Splicebio S.L. has raised €50 million (US$56.9 million) to apply its protein splicing technology to the delivery of large genes that do not fit into existing vectors. The company claims its approach will overcome the capacity constraints of adeno-associated viral vectors (AAVs), by splitting genes into parcels and reconstituting the proteins they express in vivo.
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