Results 111 to 120 of about 948 (174)

Disagreements Within the US Food and Drug Administration Regarding Approval of Novel Therapeutic Agents, 2011-2015 [PDF]

, 2020
Thirty days after a novel therapeutic agent, a new molecular entity, or original biologic is approved, the US Food and Drug Administration (FDA) must publicly disclose its approval package, including scientific reviews completed by FDA disciplines (eg ...
Herder, Matthew, MacGregor, Andrea, Ross, Joseph S., Wallach, Joshua D., Zhang, Audrey D.   +4 more
core   +1 more source

The Effect of Asfotase Alfa on Plasma and Urine Pyrophosphate Levels and Pseudofractures in a Patient With Adult-Onset Hypophosphatasia. [PDF]

JBMR Plus, 2023
Hidaka N, Murata H, Tachikawa K, Osaki K, Sekiyama T, Kinoshita Y, Kato H, Hoshino Y, Kimura S, Sunouchi T, Watanabe S, Nangaku M, Makita N, Michigami T, Ito N.   +14 more
europepmc   +1 more source

Asfotase alfa improved skeletal mineralization and fracture healing in a child with MCAHS. [PDF]

Bone, 2023
Kang M, Wu M, Crane JL.
europepmc   +1 more source

A Case of Hypophosphatasia Started Enzyme Replacement Therapy Since Babyhood Stage

Children
Background: Hypophosphatasia (HPP) is an inherited disease caused by low activity of tissue-nonspecific alkaline phosphatase. Dental characteristics include premature loss of primary teeth, enlarged pulp chambers, and enamel hypoplasia.
Tatsuya Akitomo, Noriko Niizato, Ami Kaneki, Masashi Ogawa, Taku Nishimura, Mariko Kametani, Momoko Usuda, Yuko Iwamoto, Chieko Mitsuhata, Ryota Nomura   +9 more
doaj   +1 more source

Adult-onset hypophosphatasia diagnosed after consecutive tooth loss during orthodontic treatment: a case report

Journal of Medical Case Reports
Background Adult hypophosphatasia is an uncommon inherited disorder of mineral homeostasis affecting bone. It arises from mutations within the Alkaline Phosphatase, Biomineralization Associated (ALPL) gene, which encodes tissue-nonspecific alkaline ...
Shusuke Tokuchi, Toshihiro Kawano, Edward Hosea Ntege, Makoto Murahashi, Kentaro Ide, Nobuyuki Maruyama, Risako Suzuki, Mirei Takai-Nabeta, Tsuyoshi Nabeta, Hideo Tanaka, Yusuke Shimizu, Hiroyuki Nakamura   +11 more
doaj   +1 more source

Tissue Nonspecific Alkaline Phosphatase (TNAP) Regulates Cranial Base Growth and Synchondrosis Maturation [PDF]

, 2017
Hwa K. Nam, Jin Liu, Monika Sharma, Nan E. Hatch   +3 more
core   +1 more source

Pain, quality of life, and integral management in a cohort of patients diagnosed with hypophosphatasia in Colombia

Orphanet Journal of Rare Diseases
Background Hypophosphatasia (HPP; OMIM 241510, 241500, and 146300) is a progressive metabolic, genetic disease with wide clinical heterogeneity, ranging from perinatal lethality to mild or moderate localized symptoms.
Jorge Armando Rojas Martínez, Ana María Zarante Bahamón, Luz Victoria Salazar, Andrés Felipe Morales, María Fernanda Higuera Cristancho, Juliana Villanueva Congote, Ignacio Zarante Montoya, Lina María Gómez Espitia   +7 more
doaj   +1 more source

Monoclonal antibody anti-sclerostin for treatment of pelvic insufficiency fractures in adult hypophosphatasia: A case report

Trauma Case Reports
Hypophosphatasia is a rare inherited metabolic disease leading to inhibition of bone and teeth mineralization that can be complicated by multiple insufficiency fractures.
Pierre-Emmanuel Schwab, Alicia Dessain, Joshua Milby   +2 more
doaj   +1 more source

Cutaneous lesions in the setting of hypophosphatasia

JAAD Case Reports, 2023
Nancy W. Shen, BA, Lauren G. Yi, MD, Wilson Omesiete, MD, Christina M. Peroutka, MD, Shyam S. Raghavan, MD, Kenneth E. Greer, MD   +5 more
doaj   +1 more source

Enzyme-replacement therapy in life-threatening hypophosphatasia [PDF]

, 2012
et al,, McAlister, William H, Whyte, Michael P   +2 more
core   +2 more sources