Results 231 to 240 of about 68,610 (290)

When Rare Is Not Small: Amyotrophic Lateral Sclerosis Initiatives and Therapy. [PDF]

Exploration (Beijing)
Liu Y, Xia X, Chen Y, Yang H, Chen X, Cai L, Shi B.   +6 more
europepmc   +1 more source

Societal participation in the development of orphan drugs: a systematic review. [PDF]

Front Med (Lausanne)
Akodad S, De Smedt D, Stevens H.
europepmc   +1 more source

What Do Oral Drugs Really Look Like? Dose Regimen, Pharmacokinetics, and Safety of Recently Approved Small-Molecule Oral Drugs. [PDF]

J Med Chem
Brown DG.
europepmc   +1 more source

Regulatory insights on adeno-associated virus-based gene therapies, cell- and tissue-based products, and CAR-T cell therapies: report from the 8th Asia Partnership Conference of Regenerative Medicine, April 24, 2025. [PDF]

Regen Ther
Tominaga S, Tsurumaki Y, Kagawa Y, Kito A, Miyano M, Ooka K, Kellathur SN, Hayashi S, Nomura M.   +8 more
europepmc   +1 more source

Controlled access to lumasiran in primary hyperoxaluria type 1: evaluation of a new access route for orphan drugs in the Netherlands. [PDF]

Nephrol Dial Transplant
Deesker LJ, Franssen CFM, Dorresteijn E, van de Kar NCAJ, Nurmohamed SA, Severs D, Garrelfs SF, Pisters-van Roy AAMG, Hollak CEM, Groothoff JW.   +9 more
europepmc   +1 more source

Synthesis and use of polyamine derivatives as internal mass spectrometry standards [PDF]

Dar, Sami Ullah
core  

Guiding the willingness to pay for repurposed tiratricol in the treatment of Allan-Herndon-Dudley syndrome using a cost-based-plus pricing framework

Manders EA, Keestra S, Bannenberg WJ, de Visser SJ, Hollak CE.   +4 more
europepmc   +1 more source

Innovation Policy and the Economy, Volume 3 [PDF]

Jean O. Lanjouw
core